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FDA Approves Imaavy (nipocalimab-aahu), a Neonatal Fc Receptor (FcRn) Blocker, Offering New Hope for People Living with Generalized Myasthenia Gravis (gMG)

by with No Comment New Drug Approval

Written by Priya Bhaware (M.Pharm. Pharmacology)

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The Food and Drug Administration approved Imaavy (Nipocalimab-aahu) for the treatment of generalized myasthenia gravis (gMG) in adult and paediatric patients 12 years of age and older who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive. Imaavy is a monoclonal antibody that functions as a neonatal Fc receptor (FcRn) blocker. Imaavy is developed by Johnson & Johnson and is the first treatment of its kind. It offers a new option for both adolescent and adult patients living with generalized myasthenia gravis, aiming to reduce symptoms and improve daily life.

Background and Need for New Treatments

Generalized Myasthenia Gravis (gMG) is a rare and chronic autoimmune disorder that leads to muscle weakness, making simple daily activities such as lifting your arms, chewing, swallowing, or even breathing feel extremely strenuous. The condition occurs when the immune system mistakenly attacks the neuromuscular junction and disrupts the communication pathway between nerves and muscles. This immune response, often involving harmful antibodies that target proteins like AChR or MuSK, interferes with the brain’s ability to send signals to the muscles, resulting in reduced muscle function.

Current treatments of myasthenia gravis include acetylcholinesterase inhibitors, corticosteroids, immunosuppressive drugs, plasmapheresis, plasma exchange, and intravenous immunoglobulin (IVIG). While conventional treatments can control symptoms and improve quality of life, they often come with significant side effects, invasive procedures, delayed action, flare-ups, or only temporary relief. Thus, there is a real need for more targeted and gentler treatment options, and this need and limitation have led to the development of newer, targeted therapies like monoclonal antibodies (e.g., eculizumab, ravulizumab) that aim for more precise immune modulation with fewer systemic effects, and Imaavy (nipocalimab) is the latest addition to this list of monoclonal antibodies.

Imaavy (nipocalimab-aahu): A Novel Approach

Imaavy is a targeted therapy developed to reduce specific pathogenic IgG antibodies associated with myasthenia gravis without suppressing the entire immune system. For patients who have not achieved adequate symptom control with conventional therapies, Imaavy provides a more precise mechanism of action that may improve symptom management and enhance daily functioning and quality of life.

The treatment was initially discovered by Momenta Pharmaceuticals, Inc., and subsequently licensed to Janssen Biotech, NC., a subsidiary of Johnson & Johnson, for further development and commercialization. 

Imaavy exerts its therapeutic effect by binding with high affinity to the neonatal Fc receptor (FcRn) under both acidic and neutral pH conditions. This interaction prevents the recycling of IgG antibodies including the autoantibodies implicated in myeasthenia gravis leading to their accelerated degradation and substantial reduction in total circulating IgG levels.

Clinical Trials and Approval

The U.S. Food and Drug Administration (FDA) approved Imaavy based on robust efficacy and safety data from the Phase 3 Vivacity-MG3 trial (NCT04951622), supported by findings from the ongoing Vibrance-MG pediatric study (NCT05265273).

In the Vivacity-MG3 trial, 199 adults with generalized myasthenia gravis (gMG), including both antibody-positive and antibody-negative patients inadequately controlled by existing standard therapies, were randomized to receive either nipocalimab (30 mg/kg IV loading dose followed by 15 mg/kg every two weeks) in combination with standard of care or placebo plus standard of care.

Nipocalimab demonstrated clinically meaningful benefits, including a statistically significant improvement in Myasthenia Gravis–Activities of Daily Living (MG-ADL) scores. Over the 24-week treatment period, patients receiving nipocalimab experienced an average -4.70 point improvement in MG-ADL scores, compared to -3.35 points in the placebo group. 

The clinical trial (NCT05265273), known as the Vibrance-MG study, is an ongoing Phase 2/3 open-label trial evaluating the safety, pharmacokinetics, and efficacy of nipocalimab in pediatric patients aged 2 to less than 18 years with generalized myasthenia gravis (gMG). The study aims to assess the effect of nipocalimab on total serum immunoglobulin G (IgG) levels, safety and tolerability, and pharmacokinetics in this population. Participants receive intravenous infusions of nipocalimab every two weeks during a 24-week active treatment phase, followed by a long-term extension phase and an 8-week safety follow-up. The primary outcome measures include changes in total serum IgG levels, while secondary endpoints assess improvements in Myasthenia Gravis Activities of Daily Living (MG-ADL) and Quantitative Myasthenia Gravis (QMG) scores.

The result of these two trials underscore Imaavy’s potential as a targeted and effective treatment option for adults and adolescents (ongoing) living with generalized myasthenia gravis

Safety Profile

Most reported adverse events (AEs) were mild to moderate in severity. The most commonly reported adverse reactions (occurring in ≥10% of patients) in individuals with generalized myasthenia gravis treated with Imaavy were respiratory tract infections, peripheral edema, and muscle spasms.  Serious side effects and treatment discontinuations were less common in the group receiving nivolumab than in those receiving placebo.

Patients receiving Imaavy should be monitored for infections, and administration should be delayed in those with active infections. If a serious infection develops during treatment, appropriate therapy should be initiated, and Imaavy should be withheld until the infection resolves.

Hypersensitivity reactions such as angioedema, anaphylaxis, rash, urticaria, and eczema have been reported with Imaavy. If such a reaction occurs, the infusion should be discontinued immediately, and appropriate medical management should be provided. 

In the event of a severe infusion-related reaction, treatment with Imaavy should be stopped, and appropriate intervention should be initiated. Re-administration should only be considered after carefully weighing the risks and benefits. If a mild to moderate infusion-related reaction occurs, rechallenging may be considered with close monitoring, slower infusion rates, and premedication to minimize recurrence. 

Conclusion

The approval of imaavy represents a significant advancement in addressing the complex challenges of autoimmune diseases such as generalized myasthenia gravis. By specifically targeting and reducing pathogenic IgG antibodies that interfere with nerve-to-muscle communication, Imaavy provides a novel and focused treatment option for patients whose symptoms are not adequately managed by current therapies. 

In essence, imaavy targeted mechanism of action offers new hope for individuals living with gMG, particularly those who have not responded well to conventional treatments. Furthermore, its potential applicability to a broader range of autoimmune conditions underscores its versatility and the meaningful impact it may have within the field of immunology. 

References

IMAAVY™ (nipocalimab-aahu) approved by FDA for treatment of Myasthenia Gravis (gMG) in ) in people aged 12 and older who test positive for AChR or MuSK antibodies, April 30, 2025, available from https://www.prnewswire.com/news-releases/johnson–johnson-receives-fda-approval-for-imaavy-nipocalimab-aahu-a-new-fcrn-blocker-offering-long-lasting-disease-control-in-the-broadest-population-of-people-living-with-generalized-myasthenia-gravis-gmg-302442650.html

 Dresser L, Wlodarski R, Rezania K, Soliven B. Myasthenia gravis: epidemiology, pathophysiology and clinical manifestations. Journal of clinical medicine. 2021 May 21;10(11):2235.

IMAAVY™ (nipocalimab-aahu),Highlights of Prescribing Information, available from https://www.drugs.com/imaavy.html

Nipocalimab pivotal Phase 3 trial demonstrates longest sustained disease control in FcRn class. Johnson & Johnson. June 28, 2024. https://www.jnj.com/media-center/press-releases/nipocalimab-pivotal-phase-3-trial-demonstrates-longest-sustained-disease-control-in-fcrn-class

Nipocalimab demonstrates sustained disease control in adolescents living with generalized myasthenia gravis in Phase 2/3 study. Johnson & Johnson. October 15, 2024. https://www.jnj.com/media-center/press-releases/nipocalimab-demonstrates-sustained-disease-control-in-adolescents-living-with-generalized-myasthenia-gravis-in-phase-2-3-study

Highlights of Prescribing Information, Imaavy (nipocalimab-aahu) injection,  https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/761430s000lbl.pdf

Antozzi C, Vu T, Ramchandren S, et al, Vivacity-MG3 Study Group. Safety and efficacy of nipocalimab in adults with generalised myasthenia gravis (Vivacity-MG3): a phase 3, randomised, double-blind, placebo-controlled study. Lancet Neurol. 2025 Feb;24(2):105-116. Doi: 10.1016/S1474-4422(24)00498-8. PMID: 39862879.

A Study of Nipocalimab in Children Aged 2 to Less Than 18 Years With Generalized Myasthenia Gravis, All clinical trials, NCT05265273, https://www.allclinicaltrials.com/study/NCT05265273

The article is extensively reviewed and fact-checked by the editorial team of pharmacally.com

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FDA Approval of Atzumi Marks a New Era in Migraine Treatment with SMART MucoAdhesive Delivery

by with No Comment New Drug Approval

Written by:  Utkarsha Patil (M.Pharm., Pharmacology) and Shakuntala Kawhale (M.Pharm., Pharmacology)

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The U.S. Food and Drug Administration (FDA) have approved Atzumi (dihydroergotamine mesylate) nasal powder for the acute treatment of adult migraine with or without aura, marking a major breakthrough in migraine care. This approval offers millions of migraine sufferers’ quick and efficient relief by introducing a new, non-invasive delivery method for a proven migraine medication. However the approval comes with some limitations like it is not indicated for preventive treatment of migraine or for the management of hemiplegic migraine or migraine with brainstem aura. Satsuma pharmaceuticals are known for their innovative products have been given this landmark approval by FDA. Atzumi has been developed on the SMART platform (Simple Mucoadhesive Release Technology) which uses to combines a proprietary advanced powder and device technology to simplify delivery of medicine.

Migraine and Need of the Novel Therapy

Migraine is defined as a severe, throbbing, and unilateral headache that is usually associated with photophobia (light sensitivity), phonophobia (sound sensitivity), nausea, and vomiting. It is generally considered a neurological disorder that comes in episodic attacks or presents in chronic form with or without aura.

Conventional therapy for migraine includes acute and preventive (prophylactic) treatment with various drug classes used depending on the frequency and severity of attacks. Like NSAIDs, triptans, ergotamines, and antiemetics. Preventive therapy includes anti-seizure drugs, beta-blockers, calcium channel blockers, antidepressants, and Botox treatment. 

Despite the availability of numerous medicines and these medicine have effect to minimise migraine to some extent, many patients still suffer from terrible side effects, treatment failure, or delayed relief.

Triptans, one of the most commonly prescribed classes, act as serotonin receptor agonists and work by causing vasoconstriction. However, this mechanism makes them unsuitable for individuals with cardiovascular disease. They are most effective when taken early during a migraine attack but can cause side effects such as dizziness, fatigue, and nausea. Additionally, frequent use can lead to rebound headaches or medication-overuse headaches (MOH).

NSAIDs are often used to relieve mild to moderate migraine pain, but they are typically ineffective for more severe attacks. Prolonged or frequent use can result in gastrointestinal complications, including ulcers and bleeding.

Antiemetics, which are used to alleviate nausea associated with migraines, can cause extrapyramidal symptoms such as dystonia and akathisia. They may also lead to sedation and, with long-term use, carry a risk of tardive dyskinesia.

Although Dihydroergotamine (DHE) has been used for decades to treat migraine attacks, it is associated with poor tolerability, variable absorption, and serious side effects like vasospasm and ischemia. Their use requires careful dosing to avoid toxicity.

Atzumi: A Novel Powdered Nasal Ingredient

FDA is approved Atzumi is a powdered nasal inhaler that delivers dihydroergotamine mesylate (DHE) in the form of a dry nasal powder. It provides migraineurs with a quick-acting, convenient, and needle-free alternative. Atzumi harbour various advantages over its older form of dosage and also over other therapies which includes Innovative Delivery System where it utilizes the SMART (Simple MucoAdhesive Release Technology) platform, combining advanced powder formulation with a proprietary nasal delivery device. This system ensures consistent and accurate dosing, enhancing drug absorption and patient convenience. Rapid and Sustained Absorption: Clinical studies demonstrated that Atzumi achieves rapid absorption, with mean DHE plasma concentrations of 2.0 ng/mL within approximately 15 to 20 minutes. This rapid onset is coupled with sustained plasma levels, providing prolonged relief from migraine symptoms. Improved Tolerability: Compared to injectable and liquid nasal spray forms, Atzumi’s dry powder formulation reduces issues like nasal dripping and throat irritation. Common adverse events were generally mild, including rhinitis, nausea, and altered taste. Ease of Use: The portable, single-use device allows for self-administration without the need for refrigeration, making it more accessible for patients. Non-Oral Administration: By bypassing the gastrointestinal tract, Atzumi avoids issues related to nausea and vomiting that can come with migraines, which often hinder the effectiveness of oral medications. Suitable for Triptan Non-Responders: For patients who do not respond to or cannot tolerate triptans, Atzumi provides an alternative with a different mechanism of action.

Hence, the approval of Atzumi marks an important milestone, offering a new option for the acute treatment of migraine that blends the long-established effectiveness of DHE with a user-friendly, convenient delivery system.

Ryoichi Nagata, Satsuma Pharmaceuticals said in a statement. “We believe that Atzumi will contribute to improving the quality of life of patients struggling for relief from these highly disabling problems.”

FDA Approval and Evidence from Clinical Trials

Even though DHE is in practice since long, Atzumi approval comes from rigorous clinical trials in which DHE underwent A Phase 1 pharmacokinetics (PK) trial and the Phase 3 open-label trials called ASCEND Trial, aassessed the long-term safety and tolerability of Atzumi in adults with migraine.

Phase 1 study (NCT03874832), is randomized, open-label, three-period crossover study that evaluated the pharmacokinetics, safety, and tolerability of STS101 (Atzumi), a dihydroergotamine (DHE) nasal powder, in 43 healthy adult volunteers. The study compared single doses of STS101 with intravenous (IV) DHE and DHE nasal spray, aiming to assess parameters like maximum plasma concentration (C_max) and time to reach maximum concentration (T_max). STS101 demonstrated rapid absorption, reaching effective plasma levels within 10 minutes, and achieved higher bioavailability than the DHE nasal spray. It also showed a favorable safety profile, with lower C_max than IV DHE, reducing the risk of nausea. These promising results supported the advancement of STS101 into later-phase trials, including the Phase 3 ASCEND study (NCT04406649).

The ASCEND trial (NCT04406649) was a Phase 3, open-label, multicenter study evaluating the long-term safety, tolerability, and exploratory efficacy of STS101 (Atzumi), for the acute treatment of migraine in adults aged 18 to 65 years. Participants with a history of migraine with or without aura self-administered STS101 as needed over a 12-month period, with some continuing up to 18 months. The study found that STS101 was well tolerated, with a low incidence of treatment-emergent adverse events (TEAEs), most of which were mild or moderate. Efficacy assessments showed that 36.6% of treated attacks achieved pain freedom at 2 hours post-dose, increasing to 85.5% at 24 hours. Additionally, freedom from the most bothersome symptom was reported in 54.3% of attacks at 2 hours, rising to 91.3% at 24 hours. The favorable safety and efficacy profiles observed in the ASCEND trial supported the FDA approval of Atzumi for the acute treatment of migraine with or without aura in adults.

Safety Profile

During clinical trials Atzumi shows common adverse reactions (incidence > 1%) including rhinitis, nausea, altered sense of taste, application site reactions, dizziness, vomiting, somnolence, pharyngitis, and diarrhea. However Atzumi carries a warning of serious drug interaction with strong CYP3A4 inhibitors where serious and potentially life-threatening peripheral ischemia has been linked to the combined use of dihydroergotamine and strong CYP3A4 inhibitors. These inhibitors can raise dihydroergotamine blood levels, increasing the risk of vasospasm, which may lead to cerebral or limb ischemia. Therefore, Atzumi should not be used in combination with strong CYP3A4 inhibitors.

Conclusion

Atzumi approval marks a significant advancement in migraine therapy by addressing the limitations of conventional DHE formulations. Unlike intravenous DHE, which requires administration in clinical settings and is often associated with nausea, or nasal sprays with inconsistent absorption, Atzumi delivers rapid, consistent, and sustained drug levels through a user-friendly nasal powder system. However, due to the risk of vasospasm and ischemia, Atzumi is contraindicated in patients taking strong CYP3A4 inhibitors, which can elevate DHE plasma concentrations. Overall, Atzumi provides an effective, non-invasive, and well-tolerated treatment alternative for patients seeking fast and reliable relief from migraine attacks, with clear advantages over traditional DHE therapies.

Referances

Satsuma Pharmaceuticals (2025, April 30). Satsuma Pharmaceuticals Announces U.S. FDA Approval for Atzumi™ (Dihydroergotamine) Nasal Powder for the Acute Treatment of Migraine. [News Release]. Retrieved May 2025 https://investors.satsumarx.com/2025-04-30-Satsuma-Pharmaceuticals-Announces-U-S-FDA-Approval-for-Atzumi-TM-Dihydroergotamine-Nasal-Powder-for-the-Acute-Treatment-of-Migraine

Burch R. (2024). Acute Treatment of Migraine. Continuum (Minneap Minn), 30(2), 344–363. https://doi.org/10.1212/CON.0000000000001402. PMID: 38568487.

Villar-Martinez MD, Goadsby PJ. (2022). Pathophysiology and Therapy of Associated Features of Migraine. Cells, 11(17), 2767. https://doi.org/10.3390/cells11172767. PMID: 36078174; PMCID: PMC9455236

U.S. Food & Drug Administration (FDA). (2025). Drugs@FDA: FDA-Approved Drugs – Atzumi (Dihydroergotamine Mesylate). https://www.accessdata.fda.gov

Satsuma Pharmaceuticals. (2025). Product Pipeline and Clinical Trial Data – Atzumi https://www.satsumarx.com/pipeline

Johra Khan, Lubna Ibrahim Al Asoom, et al, Genetics, pathophysiology, diagnosis, treatment, management, and prevention of migraine, Biomedicine & Pharmacotherapy, Volume 139, 2021, 111557, https://doi.org/10.1016/j.biopha.2021.111557

Albrecht D, Iwashima M, Dillon D, Harris S, Levy J. A Phase 1, Randomized, Open-Label, Safety, Tolerability, and Comparative Bioavailability Study of Intranasal Dihydroergotamine Powder (STS101), Intramuscular Dihydroergotamine Mesylate, and Intranasal DHE Mesylate Spray in Healthy Adult Subjects. Headache. 2020 Apr; 60(4):701-712. Doi: 10.1111/head.13737. Epub 2020 Jan 27. PMID: 31985049; PMCID: PMC7154716.

Tepper SJ, Albrecht D, Ailani J, Kirby L, Strom S, Rapoport AM. Long-Term (12-Month) Safety and Tolerability of STS101 (Dihydroergotamine Nasal Powder) in the Acute Treatment of Migraine: Data from the Phase 3 Open-Label ASCEND Study. CNS Drugs. 2024 Dec; 38(12):1017-1027. Doi: 10.1007/s40263-024-01118-8. Epub 2024 Oct 7. PMID: 39373843; PMCID: PMC11543709.

The article is extensively reviewed and fact-checked by editorial team of pharmacally.com

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FDA Approves PD-1 Inhibitor Penpulimab-kcqx for Advanced Nasopharyngeal Carcinoma (NPC)

by with No Comment New Drug Approval

Written By:  Samiksha Benke (M.Pharm., Pharmacology) and  Dr. Vinay Manocha (Pharm.D)

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In the landmark development, U.S. Food and Drug Administration have approved innovative PD-1 monoclonal antibody, penpulimab-kcqx, in combination with cisplatin or carboplatin and gemcitabine for the initial treatment of adult recurrent or metastatic non-keratinizing nasopharyngeal carcinoma (NPC). Penpulimab-kcqx has also been approved as a monotherapy for adult metastatic non-keratinizing NPC in patients who had progression on or after platinum-based therapy and have at least one previous line of treatment. The Food and Drug Administration has approved Penpulimab to Akeso Biopharma Co. Ltd..

Present Data and Background

Based on the WHO 2020 Global Cancer Statistics, more than 133,000 new cases of NPC occur each year worldwide, and more than 70% of the patients have locally advanced disease. Recurrent or metastatic NPC is not only associated with a poor prognosis but also with short survival. Approval of penpulimab-kcqx by the FDA will increase the number of NPC patients who can benefit from its treatment.

Nasopharyngeal carcinoma is a virulent form of squamous cell carcinoma of nasopharyngeal epithelial tissue. It is distinct from other types of head and neck cancers due to its unique epidemiology, pathology, and strong association with the Epstein-Barr virus (EBV). Penpulimab-kcqx is a PD-1 inhibitor antibody that functions to inhibit NPC tumor growth by binding to PD-1 and preventing its engagement with PD-L1 and PD-L2.

Penpulimab has earlier been approved in China for multiple indications, including as a first-line treatment along with chemotherapy for locally advanced or metastatic squamous non-small cell lung cancer, third-line treatment of relapsed or refractory classical Hodgkin’s lymphoma, and third-line treatment of recurrent or metastatic NPC.

Penpulimab: A Novel Approach

Penpulimab, also referred to as AK105, is a human IgG1 monoclonal antibody targeting PD-1. It features a distinct Fc region mutation that eliminates interactions with Fc gamma receptors (FcƴRs) and the complement component C1 system, effectively preventing antibody-dependent cellular cytotoxicity (ADCC), antibody-dependent cellular phagocytosis (ADCP), and complement-dependent cytotoxicity (CDC). These functions are typically triggered by binding to FcγR-IIIa, FcγR-Ia, and C1q, respectively. Moreover, the IgG1 backbone provides structural stability, helping to reduce immune escape.

Clinical trials and approval

The approval of penpulimab-kcqx as a first-line treatment was based on the randomized, double-blind AK105-304 trial (ClinicalTrials.gov Identifier: NCT04974398), and the approval of penpulimab-kcqx as a single agent for NPC was based on the open-label, single-arm AK105-202 study (ClinicalTrials.gov Identifier: NCT03866967).

The AK105-304 trial was a Phase III, multi-center, randomized, double-blind, placebo-controlled study designed to assess the efficacy of Penpulimab (AK105) in combination with chemotherapy as a first-line treatment for non-keratinizing nasopharyngeal carcinoma (NPC). A total of 291 patients with histologically confirmed NPC who had not received prior systemic therapy for metastatic disease were enrolled and randomized in a 1:1 ratio to receive Penpulimab plus chemotherapy or placebo plus chemotherapy.

The chemotherapy regimen consisted of cisplatin (80 mg/m²) and gemcitabine (1,000 mg/m²), administered every three weeks for up to six cycles. Patients in the experimental arm received Penpulimab (200 mg IV) every two weeks, followed by Penpulimab monotherapy as maintenance. In the control arm, patients were allowed to switch to Penpulimab monotherapy upon disease progression.

Penpulimab demonstrated a 55% reduction in the risk of disease progression or death. The median progression-free survival (PFS) was 9.6 months in the Penpulimab group compared to 7.0 months in the placebo group. At the 12-month mark, 31% of patients in the Penpulimab arm remained progression-free, versus only 11% in the placebo arm.

The AK105-202 trial was a Phase II, open-label, single-arm study involving 125 patients with metastatic or unresectable non-keratinizing nasopharyngeal carcinoma (NPC) who had experienced disease progression following platinum-based chemotherapy and at least one additional prior line of therapy. Participants received penpulimab-kcqx monotherapy for up to 24 months, or until disease progression or unacceptable toxicity occurred.

The primary endpoints objective response rate (ORR) and duration of response (DOR) were assessed by an independent radiology review committee based on RECIST v1.1 criteria. The results showed a partial response in 27% of patients, with an ORR of 28% the median DOR was not reached and 46% of responders maintained a DOR of at least 12 months.

Safety profile

Penpulimab, when used in combination with cisplatin, carboplatin, and gemcitabine, was most commonly associated with adverse events such as vomiting, nausea, hypothyroidism, constipation, decreased appetite, weight loss, cough, COVID-19, infections, fatigue, rash, and fever, each reported in at least 20% of patients.

For patients treated with penpulimab as a single agent, the most frequently observed adverse events, affecting at least 20% of individuals, were musculoskeletal pain and hypothyroidism.

Serious and potentially fatal adverse drug reactions, including pneumonitis, colitis, septic shock, and hepatitis, were reported in approximately 1% of cases. Immune-mediated toxicities, particularly pneumonitis and colitis, require careful monitoring.

Overall, penpulimab exhibited a manageable safety profile, with the majority of adverse events classified as mild to moderate in severity. 

Impact and Future Viewpoint

This FDA approval provides a new immunotherapy option for patients with advanced NPC, potentially improving outcomes and offering hope for those with limited treatment alternatives. The approval also emphasizes the importance of continuing research and development in rare cancers, aiming to enhance patient care and survival rates.

Future research will explore long-term benefits, optimal uses, and potential drug combinations, including targeted agents or radiation therapy. Its global approval will increase patient access to this promising immunotherapy, offering new hope for NPC treatment.

References

Akeso Announces FDA Approval for Penpulimab-kcqx in Two BLA Indications for Comprehensive Treatment of Advanced Nasopharyngeal Carcinoma, 25 April 2025, Akesobio, https://www.akesobio.com/en/media/akeso-news/250425/

FDA approves penpulimab-kcqx for non-keratinizing nasopharyngeal carcinoma, US Food and Drug Administration, 24 April 2025 https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-penpulimab-kcqx-non-keratinizing-nasopharyngeal-carcinoma

Chen X, Wang W, Zou Q, et al, Penpulimab, an anti-PD-1 antibody, for heavily pretreated metastatic nasopharyngeal carcinoma: a single-arm phase II study. Signal Transduct Target Ther. 2024 Jun 19;9(1):148. Doi: 10.1038/s41392-024-01865-6. PMID: 38890298; PMCID: PMC11189389.

Song Y, Zhou K, Jin C, et al, Penpulimab for Relapsed or Refractory Classical Hodgkin Lymphoma: A Multicenter, Single-Arm, Pivotal Phase I/II Trial (AK105-201). Front Oncol. 2022 Jul 7;12:925236. doi: 10.3389/fonc.2022.925236. PMID: 35875118; PMCID: PMC9301139. https://pmc.ncbi.nlm.nih.gov/articles/PMC9301139/

Akeso announces FDA approval for penpulimab-kcqx in two BLA indications for comprehensive treatment of advanced nasopharyngeal carcinoma. News release. Akeso. April 24, 2025. https://www.prnewswire.com/news-releases/akeso-announces-fda-approval-for-penpulimab-kcqx-in-two-bla-indications-for-comprehensive-treatment-of-advanced-nasopharyngeal-carcinoma-302437965.html.

Chaosu Hu, Xiaozhong Chen, Tingting Xu, et al, Penpulimab versus placebo in combination with chemotherapy as first-line treatment for recurrent or metastatic nasopharyngeal carcinoma: A global, multicenter, randomized, double-blind, phase 3 trial (AK105-304) [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2025; Part 2 (Late-Breaking, Clinical Trial, and Invited Abstracts); 2025 Apr 25-30; Chicago, IL. Philadelphia (PA): AACR; Cancer Res 2025;85(8_Suppl_2):Abstract nr CT011.

The article is extensively reviewed and fact-checked by the editorial team of pharmacally.com

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FDA Approves Zevaskyn (Prademagene Zamikeracel) for Recessive Dystrophic Epidermolysis Bullosa (RDEB): Gene Therapy Targets COL7A1 Gene and Boosts Type VII Collagen (C7)

by with No Comment New Drug Approval

Written by:  Rikesh Dighore (M Pharm., Pharmacology) , and  Soniya Hajare (M Pharm.., Pharmacology)

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For the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB), the FDA has approved Zevaskyn (prademagene zamikeracel), the first autologous cell-based gene therapy. The treatment of RDEB, a rare and disabling genetic skin condition marked by fragile skin, prone to blistering and recurrent wounds, has advanced significantly with this approval. Abeona Therapeutics has developed Zevaskyn, a novel cell-based gene therapy scientifically known as prademagene zamikeracel (previously referred to as pz-cel or EB-101). By directly addressing the genetic mutation responsible for the disease, it represents a significant advancement in the treatment of RDEB. By using genetically modifying skin cells to create type VII collagen, Zevaskyn provides a novel therapeutic approach that improves wound healing and quality of patient’s life.

About Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Recessive Dystrophic Epidermolysis Bullosa (RDEB) is one of the subtypes of various Epidermolysis Bullosa. Epidermolysis Bullosa is the heterogeneous group of inherited mechanobullous disorders triggered by mutations in genes that encode structural proteins in the skin. RDEB involves mutations in the COL7A1 gene, which codes for type VII collagen (C7). Type II collagen protein is essential to the skin’s structural integrity. The mutation in this gene leads to anomalous synthesis of type II collagen or faulty compilation of the protein into anchoring fibrils, which results in poor epidermal-dermal cohesion.

As a result skin is become very delicate because the layers do not attach correctly without this protein. Hence, even with little contact or impact, people with RDEB experiences open wounds and persistent blistering. A complication which arises due to this condition includes severe infections, malnourishment, scarring, joint contractures, and an elevated risk of aggressive squamous cell carcinoma (SCC). The disease affects both the skin and internal mucous membranes. Many patients have a shorter life expectancy and frequently do not live past their 30s or 40s.

However, there is currently no cure for RDEB. Symptomatic treatment which includes pain management, infection prevention and control, and careful wound care, has been the main focus of standard care. Despite being necessary, these supportive treatments are quite strenuous, necessitate intensive daily interventions, and fail to address the underlying genetic problem. Additionally, current topical treatments like Vyjuvek which was approved for Dystrophic Epidermolysis Bullosa are insufficient for large, chronic, or deep wounds that considerably lower quality of life and raise the risk of death, even though they work well for minor, superficial wounds.

Hence, a targeted, long-lasting treatment alternative was in urgent need as evident from the significant morbidity and mortality rates linked to RDEB.

Zevaskyn (prademagene zamikeracel): A Novel Approach

Zevaskyn (prademagene zamikeracel) received FDA approval in April 2025, tackled a critical gap in treatment. This autologous, ex vivo gene therapy uses a retroviral vector to modify a patient’s own keratinocytes, allowing them to produce functional type VII collagen. The corrected cells are then grafted onto the patient’s chronic wounds. Unlike topical treatments that require ongoing application, Zevaskyn is designed as a one-time therapy offering long-lasting effects by providing permanent structural repair to the skin.

By enhancing skin integrity, reducing infection rates, and maybe slowing the growth of skin malignancies, this therapeutic breakthrough not only lessens the burden of care and discomfort, but also has the potential to extend life. Zevaskyn thus fills a long standing medical gap in the treatment of RDEB and marks a significant breakthrough.

Clinical Trials and FDA Approval

The FDA approval of ZEVASKYN (prademagene zamikeracel) was primarily based on the pivotal Phase 3 VIITAL™ study (NCT04227106). This multicenter, randomized, intrapatient-controlled trial evaluated the efficacy of a single application of ZEVASKYN in treating large, chronic wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB). Patients with RDEB received skin grafts made of their own genetically modified cells as part of this randomized, controlled study. The study met its two co-primary endpoints.

Wound Healing: 81% of wounds treated with ZEVASKYN achieved 50% or more healing at six months, compared to 16% in control wounds treated with standard care (P<0.0001).

Pain Reduction: Patients reported significant pain reduction from baseline, as assessed by the Wong-Baker FACES scale.

Additionally, the FDA considered data from a Phase 1/2a study (NCT01263379), demonstrating long-term improvement in treated sites over a median follow-up of 6.9 years. These studies collectively supported the approval of ZEVASKYN for treating wounds in adult and pediatric patients with RDEB.

Rowan is a 3-year-old girl living with Recessive Dystrophic Epidermolysis Bullosa in her daily life.

Source: EB Research Partnership (YouTube) _ Rowan’s Story 

Safety Profile

In clinical trials, Zevaskyn has shown a good safety profile. Localized to the treatment site, the most often reported side effects included itching and soreness during the grafting process. Significantly, the trials showed no significant side effects that were directly linked to the treatment.

The risk of immunological rejection and other issues related to allogeneic transplants is decreased when the patient’s own cells are used in the treatment.

Impact

Zevaskyn’s approval gives patients with RDEB new hope and marks a major advancement in the field of gene therapy. Zevaskyn has the potential to change the standard of care and enhance the lives of those badly affected by this debilitating illness by offering a medication that targets the underlying cause of the illness and not just symptoms.

Additionally, Zevaskyn enhances current treatments like Vyjuvek (beremagene geperpavec), a topical gene therapy that was authorized in 2023 to treat minor wounds in people with DEB. The dual-action armament in treatments like Vyjuvek, treating smaller lesions, and Zevaskyn, targeting larger, chronic wounds, enables a more thorough approach to disease management.

Conclusion

Zevaskyn’s FDA clearance represents a major advancement in the management of recessive dystrophic epidermolysis bullosa a rare and devastating genetic skin disorder that has long imposed a severe burden on patients and families. ZEVASKYN, the first approved topical gene therapy for this condition, offers new hope by directly addressing the underlying genetic defect.

Its approval was backed by compelling data from the pivotal Phase 3 VIITAL™ trial, which showed significant improvements in wound healing and pain reduction two of the most debilitating aspects of RDEB. Unlike traditional therapies focused only on symptom management, ZEVASKYN delivers a corrected COL7A1 gene to the skin, promoting true biological repair.

Zevaskyn may open the door to more personalized and efficient treatments for uncommon genetic illnesses as research advances and availability increases.

References

Zevaskyn approval letter, US Food and Drug Administration, available from https://www.fda.gov/media/186513/download?attachment

Bischof J, Hierl M, Koller U. Emerging Gene Therapeutics for Epidermolysis Bullosa under Development. Int J Mol Sci. 2024 Feb 13;25(4):2243. doi: 10.3390/ijms25042243. PMID: 38396920; PMCID: PMC10889532.

Luis Soro, Do, Cynthia Bartus, Stephen Purcell et al, Recessive Dystrophic Epidermolysis Bullosa A Review of Disease Pathogenesis and Update on Future Therapies, May 2015, Volume 8, Number 5, The Journal of Clinical and Aesthetic Dermatology. https://pmc.ncbi.nlm.nih.gov/articles/PMC4445895/pdf/jcad_8_5_41.pdf

Hon KL, Chu S, Leung AKC. Epidermolysis Bullosa: Pediatric Perspectives. Curr Pediatr Rev. 2022; 18(3):182-190. Doi: 10.2174/1573396317666210525161252. PMID: 34036913.

Gurevich I, Agarwal P, Zhang P, Dolorito JA, Oliver S, Liu H, Reitze N, Sarma N, Bagci IS, Sridhar K, Kakarla V, Yenamandra VK, O’Malley M, Prisco M, Tufa SF, Keene DR, South AP, Krishnan SM, Marinkovich MP. In vivo topical gene therapy for recessive dystrophic epidermolysis bullosa: a phase 1 and 2 trial. Nat Med. 2022 Apr;28(4):780-788. Doi: 10.1038/s41591-022-01737-y. Epub 2022 Mar 28. PMID: 35347281; PMCID: PMC9018416.

Abeona Therapeutics (2025). Zevaskyn (prademagene zamikeracel): Product Information and VIITAL Study Results. https://www.abeonatherapeutics.com

Koutsoukos SA, Bilousova G. Highlights of Gene and Cell Therapy for Epidermolysis Bullosa and Ichthyosis. Dermatol Ther (Heidelb). 2024 Sep;14(9):2379-2392. Doi: 10.1007/s13555-024-01239-4. Epub 2024 Aug 7. PMID: 39112824; PMCID: PMC11393223.

Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB), ClinicalTrials.gov ID NCT04227106, https://clinicaltrials.gov/study/NCT04227106

The article is extensively reviewed and fact-checked by the editorial team of pharmacally.com

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FDA Grants Accelerated Approval to Avmapki Fakzynja Co-Pack for KRAS-Mutated Low Grade Serous Ovarian Cancer (LGSOC) Targeting RAS/MAPK Pathway

by with No Comment New Drug Approval

Written By: Shital Gaikwad (M.Pharm Pharmacology) and Shital Doifode (M.Pharm Pharmacology)

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In image ovarian cancer cells (Source: Freepik.com)

In a significant progress in ovarian cancer treatment, the U.S. Food and Drug Administration (FDA) has granted accelerated approval to Avmapki Fakzynja Co-Pack (avutometinib capsules and defactinib tablets) for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy. This approval represents the first and only FDA-approved treatment specifically for this rare subtype of ovarian cancer, marking a significant step forward in the fight against RAS/MAPK pathway-driven tumors. The approval is granted to Verastem Oncology, a biopharmaceutical company dedicated in the advancement of new treatments for patients with RAS/MAPK pathway-determined cancers. The FDA confirmed that this approval is under the accelerated approval, dependent on tumor response rate and duration of response; however, continuous approval for this indication will be subject to the results of the confirmatory trial. The FDA has also granted this combination Orphan Drug Designation.

Background and Need for New Treatments

Low-grade serous ovarian cancer (LGSOC) is a rare and recurrent form of epithelial ovarian cancer that mainly affects younger women and has a poor response to conventional chemotherapy. Approximately 30%–60% of LGSOC tumors carry KRAS mutations, activating the RAS/MAPK pathway and promoting uncontrolled tumor growth. KRAS mutation leads to continuous activation of the RAS/MAPK pathway.

There has been no FDA-approved treatment particularly targeting KRAS-mutated recurrent LGSOC, and patients have had limited treatment options like surgery, chemotherapy hormonal However each treatment options had certain limitations like low sensitivity to chemotherapy, high recurrence rate, toxicity of hormonal therapy and lacking of options of personalising treatment. This need highlighted the urgent demand for a targeted, effective, and tolerable treatment for this patient population.

Avmapki Fakzynja Co-Pack: A First-in-Class Combination

Avmapki Fakzynja Co-Pack is a novel oral combination therapy that includes:

Avutometinib: Avutometinib is an inhibitor of MEK1 that promotes the formation of inactive RAF/MEK complexes, thereby blocking RAF-mediated phosphorylation of MEK1/2. RAF and MEK are key components in the RAS/RAF/MEK/ERK (MAPK) signaling pathway. Avutometinib suppresses phosphorylation of MEK1/2 and ERK1/2, as well as the growth of tumor cell lines with KRAS mutations. Additionally, avutometinib treatment leads to an increase in phosphorylated focal adhesion kinase (FAK) levels in cancer cells.

Defactinib: Defactinib is a selective inhibitor of focal adhesion kinase (FAK) and proline-rich tyrosine kinase 2 (Pyk2), both part of the FAK family of non-receptor tyrosine kinases. It has been shown to block FAK autophosphorylation in cancer cells in vitro and in mouse xenograft models.

This synergistic effect of both drugs leads to enhanced tumor response by concurrently inhibiting both tumor cell proliferation and survival mechanisms, making it a breakthrough option for KRAS-mutant LGSOC.

Clinical Trials and FDA Approval Basis

The accelerated approval of Avmapki Fakzynja Co-Pack was based on the results of the Phase II RAMP 201 clinical trial, which evaluated the safety and efficacy of avutometinib, a RAF/MEK inhibitor, alone and in combination with defactinib, a FAK inhibitor, in patients with recurrent low-grade serous ovarian cancer (LGSOC), particularly those with KRAS mutations.

Study Design

RAMP 201 (NCT04625270) was a multicenter, randomized, open-label Phase II study. It enrolled patients aged 18 years or older with histologically confirmed LGSOC or peritoneal cancer, measurable disease per RECIST 1.1 criteria, and disease progression or recurrence following at least one prior systemic therapy in the metastatic setting. The trial included patients with both KRAS-mutated and wild-type tumors. The primary objective was to assess the overall response rate (ORR) of the treatments.

Key findings from the trial:

Efficacy: In patients receiving the combination of avutometinib (3.2 mg twice weekly) and defactinib (200 mg twice daily), the confirmed ORR was 31%, with a complete response rate of 2% and a partial response rate of 29%. Among patients with KRAS-mutated tumors, the ORR was higher at 44%, compared to 17% in those with KRAS wild-type tumors.

Progression-Free Survival (PFS): The median PFS for the combination therapy was reported as 12.9 months, with longer durations observed in patients carrying KRAS mutations.

Confirmatory Trials

The continuous approval of this combination therapy is reliant on confirmatory evidence from the ongoing Phase III RAMP 301 trial, which will further evaluate clinical benefit in a broader population, including patients with and without KRAS mutations.

If the clinical benefit is confirmed, this would harden the combination as the standard of care for this difficult-to-treat cancer.

Side effects

Common (≥25%) Adverse Reactions (including lab abnormalities):

Elevated creatine phosphokinase, nausea, fatigue, increased AST and ALT, rash, diarrhea, musculoskeletal pain, edema, low hemoglobin, high bilirubin and triglycerides, low lymphocytes, abdominal pain, dyspepsia, acneiform dermatitis, vitreoretinal disorders, high alkaline phosphatase, stomatitis, itching, visual impairment, low platelets, constipation, dry skin, shortness of breath, cough, urinary tract infection, and low neutrophils.

Impact

The accelerated approval of Avmapki Fakzynja marks an important step in personalized oncology, especially for women fighting recurrent LGSOC with KRAS mutations, in view of few effective conventional treatment options.

This is the first FDA-approved therapy targeting KRAS-mutated LGSOC, reflecting a significant advancement in precision medicine. It also validates the potential of combining MEK and FAK inhibition to address tumor growth and resistance mechanisms.

Expert Opinion from Investigators

Dr. Rachel Grisham of Memorial Sloan Kettering Cancer Center and Weill Cornell Medical College, New York, and lead investigator of the RAMP 301 study, stated:

“This approval marks a much-needed therapeutic option and establishes this combination as the new standard of care for women with recurrent LGSOC harbouring a KRAS mutation.”

Prof. Susana Banerjee of The Royal Marsden NHS Foundation Trust and Institute of Cancer Research, National Cancer Research Institute (NCRI), London also noted:

“To see this combination advance from early trials to become the first-ever FDA-approved therapy for LGSOC is inspiring and opens a new chapter in treating RAS/MAPK-pathway-driven cancers.”

Conclusion

The FDA’s accelerated approval of Avmapki Fakzynja Co-Pack introduces a much-needed targeted therapy for patients with KRAS-mutated recurrent low-grade serous ovarian cancer. With its novel dual-mechanism approach and promising clinical results, this combination therapy offers new hope for improving survival and quality of life in a population with limited options for LGSOC. However, its continuous approval and use depend on the success of the ongoing phase III trial. As precision oncology advances, the success of treatments like Avmapki underscores the importance of genetic profiling and personalized treatment strategies in overcoming rare and resistant cancers.

References

U.S. Food and Drug Administration. (2025, May 8). FDA grants accelerated approval to the combination of avutometinib and defactinib for KRAS-mutated recurrent low-grade serous ovarian cancer. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-combination-avutometinib-and-defactinib-kras-mutated-recurrent-low

Verastem Oncology. (2025, May 8). Verastem Oncology announces FDA approval of Avmapki Fakzynja Co-Pack for the treatment of KRAS-mutated recurrent low-grade serous ovarian cancer [Press release]. https://verastem.com/news/fda-approval-avmapki-fakzynja

ClinicalTrials.gov. (n.d.). A study of avutometinib (VS-6766) and defactinib in patients with recurrent low grade serous ovarian cancer (RAMP 201) (NCT04625270). National Library of Medicine. https://clinicaltrials.gov/study/NCT04625270

National Cancer Institute. (2024). Low-grade serous carcinoma of the ovary. https://www.cancer.gov/pediatric-adult-rare-tumor/rare-tumors/rare-gynecologic-tumors/low-grade-serous-carcinoma

Highlights of Prescribing Information, Avmapkitm Fakzynjatm Co-Pack, available from https://www.verastem.com/pdf/avmapki-fakzynja-co-pack-full-prescribing-information.pdf

FDA Grants Accelerated Approval to Avmapki Fakzynja Co-Pack, Drugs.com,available from https://www.drugs.com/newdrugs/fda-grants-accelerated-approval-avmapki-fakzynja-co-pack-avutometinib-capsules-defactinib-kras-6516.html

Susana N. Banerjee et al. ENGOT-ov60/GOG-3052/RAMP 201: A phase 2 study of VS-6766 (RAF/MEK clamp) alone and in combination with defactinib (FAK inhibitor) in recurrent low-grade serous ovarian cancer (LGSOC).. JCO 40, TPS5615-TPS5615(2022). DOI:10.1200/JCO.2022.40.16_suppl.TPS5615

The article is extensively reviewed and fact-checked by the editorial team of the pharmacally.com

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Japan First-in-the-World to approve Elevidys (Delandistrogene Moxeparvovec), one-of-the Costliest Gene Therapy Drug for Duchenne Muscular Dystrophy (DMD) in Children less than 4 years of age

by with No Comment New Drug Approval

Written By: Pragati Ekamalli (B.Pharm)

Reviewed By: Vikas Londhe M.Pharm (Pharmacology)

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Source: Freepik.com

In a groundbreaking step forward for genetic medicine, Japan has become the first country in the world to approve the use of Elevidys (delandistrogene moxeparvovec), a gene therapy created by Sarepta Therapeutics, for children under 4 years old diagnosed with Duchenne muscular dystrophy (DMD). DMD is a rare and life-threatening genetic disorder that leads to the progressive weakening and loss of muscle function, typically appearing in early childhood and worsening over time. Elevidys works by delivering a modified gene to help produce a version of the dystrophin protein, which is missing or defective in children with DMD. This approval represents a major milestone in the global effort to treat genetic disorders and could bring new hope to families affected by DMD. It also highlights Japan’s leadership in adopting cutting-edge medical treatments that may slow disease progression and improve quality of life for young patients.

Understanding Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a rare, inherited disorder that mainly affects boys due to its X-linked pattern of inheritance, meaning the faulty gene is carried on the X chromosome. The condition results from mutations in the DMD gene, which is responsible for producing dystrophin a protein critical for maintaining the strength and stability of muscle fibers. In the absence of functional dystrophin, muscles are unable to protect themselves from everyday stress, leading to ongoing damage, muscle fibres breakdown, and replacement by fat and scar tissue. Symptoms usually begin in early childhood, often with difficulty walking, running, or climbing stairs. As the disease progresses, most affected children lose their ability to walk by the age of 12 to 14. In later stages, DMD can lead to serious complications involving the heart and lungs, often reducing life expectancy into the twenties or thirties without advanced medical care.

Understanding Dystrophin Protein

Dystrophin is large structural protein (427kDa) present in cytoplasmic face of the sarcolemma. Dystrophin protein plays a crucial role in maintaining the integrity and function of muscle cells including skeletal muscle and cardiac muscle.

Dystrophin is act like an anchor which link internal cytoskeleton of a muscle fibre to the surrounding extracellular matrix by dystrophin-glycoprotein complex. This complex stabilizes muscle fibre during contraction. Dystrophin protein is coded with DMD gene on X-Chromosome and it is one of the largest human genes. Any mutation or deletion of DMD genes affects the dystrophin protein production, either less or completely diminished.

Elevidys: A Novel Gene Therapy Approach

Elevidys is an innovative gene therapy that uses an adeno-associated virus (AAV) as a delivery system to transport genetic material into the body. Specifically designed for Duchenne muscular dystrophy (DMD), Elevidys delivers a shortened but functional version of the dystrophin gene, called micro-dystrophin, directly into muscle cells. Although smaller than the full-length gene, micro-dystrophin includes the critical domains needed for the protein to support muscle fibre structure and function. By enabling cells to produce this essential protein, Elevidys targets the root cause of DMD — the absence of functional dystrophin. Administered as a single intravenous infusion, this therapy offers the promise of a one-time treatment that may slow disease progression and improve muscle strength, potentially transforming the standard of care for children living with DMD.

Japan’s Regulatory Approval

On August 14, 2024, Chugai Pharmaceutical Co., Ltd., a member of the Roche Group, submitted a regulatory application to Japan’s Ministry of Health, Labour and Welfare (MHLW) seeking approval for Elevidys, a gene therapy for Duchenne muscular dystrophy (DMD). The application specifically targeted ambulatory boys aged 3 to 7 years who do not have deletions in exons 8 and/or 9 of the DMD gene and who are not immune to the AAVrh74 viral vector used to deliver the treatment. This submission was supported by data from the global Phase III EMBARK study, which evaluated the safety and effectiveness of Elevidys. The trial did not meet its primary endpoint improving motor function as measured by the North Star Ambulatory Assessment (NSAA), however clinically meaningful benefits were observed in key secondary outcomes, including faster time to rise from the floor and improved 10-meter walk speed. Despite the mixed results, Japan’s MHLW granted priority review to the application and ultimately approved Elevidys for use in children less than 4 years of age. With this decision, Japan became the first country to authorize Elevidys for this younger age group, reflecting its proactive approach to approving innovative therapies for rare and serious diseases like DMD. This landmark approval offers new hope for patients and families affected by this devastating condition.

Global Context

Before Japan’s historic approval, the U.S. Food and Drug Administration (FDA) granted Elevidys accelerated approval in June 2023 for ambulatory children aged 4 through 5 years with Duchenne muscular dystrophy (DMD). The FDA’s decision was based on evidence that the therapy successfully prompted the production of micro-dystrophin, the shortened version of the dystrophin protein essential for muscle health. However, this approval was conditional, with continued authorization depending on the confirmation of meaningful clinical benefits in ongoing follow-up trials. Japan’s more inclusive approval for children under 4 years old not only marks a significant expansion of access to this gene therapy but also sets a global precedent. It signals to other regulatory agencies the potential value of early intervention in DMD. As new clinical data emerges and confirmatory studies continue, more countries may follow suit, potentially reshaping the global treatment landscape for this devastating disease and offering hope for earlier and more effective management of DMD in young patients.

Conclusion

The approval of Elevidys in Japan for children younger than 4 years of age marks a major breakthrough in the treatment of Duchenne muscular dystrophy (DMD). Unlike traditional therapies that mainly manage symptoms, Elevidys directly targets the genetic root of the disease by introducing a functional version of the dystrophin gene. This approach offers the potential not just to slow the progression of muscle degeneration, but to meaningfully alter the course of the disease, particularly when administered early in life. The decision by Japan’s health authorities highlights the critical role of early intervention and paves the way for improving long-term outcomes for children affected by DMD. Moreover, this milestone emphasizes the value of ongoing scientific research, clinical trials, and international collaboration in developing transformative therapies for rare and life-threatening conditions.

References

1. Sarepta Therapeutics Announces Approval in Japan of ELEVIDYS, a Gene Therapy to Treat Duchenne Muscular Dystrophy, 13 May 2025, Sarepta Therapeutics https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-approval-japan-elevidys-gene

2. A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) (EMBARK), NCT05096221, ClinicalTrials.gov, available from https://clinicaltrials.gov/study/NCT05096221

3. Roche announces new results from EMBARK demonstrating significant sustained benefits of Elevidys in ambulatory individuals with Duchenne muscular dystrophy (DMD), 27 January 2025, https://www.roche.com/media/releases/med-cor-2025-01-27

4. Gao QQ, McNally EM. The Dystrophin Complex: Structure, Function, and Implications for Therapy. Compr Physiol. 2015 Jul 1; 5(3):1223-39. Doi: 10.1002/cphy.c140048. PMID: 26140716; PMCID: PMC4767260.

5. Venugopal V, Pavlakis S. Duchenne Muscular Dystrophy. [Updated 2023 Jul 10]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2025 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK482346/

6. Chugai Receives Regulatory Approval for “ELEVIDYS” as a Gene Therapy Product for Duchenne Muscular Dystrophy in Japan, 13 May 2025, Chugai Pharmaceuticals,https://www.chugaipharm.co.jp/english/news/detail/20250513181500_1160.html

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“Microplastics in Human Tissues: Insights from Nature Medicine and NEJM Studies”

by with No Comment Research

Written By Lavanya Chavhan B.Pharm

Reviewed and Fact Checked By Vikas Londhe M.Pharm (Pharmacology)

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Source: Freepik.com

Microplastics and nanoplastics (MNPs) tiny particles resulting from the degradation of plastic materials have rapidly become a focus of global health research. Two landmark studies published in Nature Medicine and The New England Journal of Medicine (NEJM) offer critical insights into the bioaccumulation and health consequences of these particles in human tissues. Together, they present an urgent call to re-evaluate the long-term implications of environmental plastic pollution.

Brain Accumulation and Neurological Concerns: Findings from Nature Medicine

A study published in Nature Medicine (April 2025) examined the presence of MNPs in human tissues, with a focus on liver, kidney, and brain samples from autopsies conducted in 2016 and 2024. The researchers used advanced analytical techniques including pyrolysis gas chromatography mass spectrometry (Py-GC/MS), electron microscopy, and Fourier-transform infrared spectroscopy to identify and quantify plastic particles.

Key Findings

Ubiquitous Presence: MNPs were detected in all three organs, but brain tissues exhibited the highest concentrations, 7 to 30 times greater than liver or kidney.

Polyethylene (PE) Dominance: PE accounted for approximately 75% of the plastic content in brains.

Temporal Trends: There was a significant increase in plastic concentrations between 2016 and 2024, reflecting rising environmental exposure.

Dementia Link: Brains of individuals with documented dementia had markedly higher levels of MNPs up to 26,076 µg/g suggesting a possible connection with neurodegenerative processes

Tissue Localization: Nanoplastics were found in brain parenchyma, immune cells, and cerebrovascular walls, raising questions about blood-brain barrier integrity and inflammatory responses.

Conclusion: Although the study does not establish causality, it strongly suggests that chronic exposure to MNPs may be associated with neurological vulnerability. The findings warrant deeper exploration into how plastic particles reach and affect brain tissue, particularly in individuals with compromised neurological health.

Cardiovascular Risks and Atheroma Infiltration: NEJM Study

The NEJM study (March 2024) provided the first direct clinical evidence linking MNPs to cardiovascular risk. It was a multicenter, prospective study involving 304 patients undergoing carotid endarterectomy for asymptomatic carotid artery disease.

Key Findings:

MNP Detection: In 58.4% of patients, polyethylene was found within atherosclerotic plaques. Polyvinyl chloride was also detected in 12.1%.

Nanoplastics Inside Plaques: Electron microscopy revealed jagged-edged particles (<1 µm) embedded in foam cells and extracellular plaque matrix.

Increased Cardiovascular Events: Over a 34-month follow-up, patients with MNP-positive plaques were at 4.5 times greater risk of myocardial infarction, stroke, or death compared to those without detectable plastics.

Inflammation Correlation: Elevated levels of interleukin-1β, IL-6, TNF-α, and CD68 were observed in MNP-positive plaques, suggesting heightened inflammatory responses.

Conclusion: The presence of MNPs in vascular tissues appears to be more than incidental. It correlates with both histological inflammation and clinically significant cardiovascular outcomes. This raises red flags about MNPs as emerging risk factors in heart and vascular diseases.

Implications for Human Health and Future Research

The convergence of findings from brain and vascular tissues paints a concerning picture:

Bioaccumulation is Organ-Specific: MNPs preferentially accumulate in certain tissues, potentially due to local physiological characteristics like vascular density or barrier permeability.

Health Impact is Multisystemic: While one study implicates MNPs in neurodegeneration, the other links them to cardiovascular events two of the most pressing areas in public health.

Mechanistic Gaps Remain: Both studies acknowledge the limitations in establishing causality. The exact pathways of MNP absorption, translocation, and clearance in humans remain poorly understood.

Another small study presented in American Heart Association conference

At the American Heart Association’s Vascular Discovery Scientific Sessions held in Baltimore on April 22, 2025, researchers presented compelling evidence linking microplastics to vascular disease. The study, led by Dr. Ross Clark from the University of New Mexico, analyzed carotid artery plaques from individuals who had experienced strokes, mini-strokes, or transient vision loss. Findings revealed that these plaques contained microplastics at concentrations 51 times higher than those in healthy arteries. Notably, polyethylene and polyvinyl chloride were the predominant plastics detected. While the study did not establish a direct causal relationship, it highlighted a strong association between microplastic accumulation and symptomatic arterial disease, underscoring the need for further research into the potential health impacts of environmental plastic exposure.

Conclusion

As plastic production continues to grow rapidly across the globe, recent studies serve as a strong warning that we must take urgent action. This includes putting better regulations in place, increasing scientific research, and raising public health awareness. Tiny plastic particles known as microplastics and nanoplastics are now found in the air we breathe, the food we eat, and even in our bodies. These particles may carry harmful chemicals and can travel through the blood stream to vital organs. Over time, long term exposure to them could increase the risk of serious health problems, including disease of the brain, heart, lungs and more.

To truly understand how these plastics affect our health, we need experts from many different fields to work together. Toxicologists can study how the plastics interact with our cells, neurologists can explore their impact on the brain and nervous system, cardiologists can look into heart-related effects, and environmental scientists can track how plastics move through ecosystems and into our bodies. Only through this kind of teamwork can we begin to understand plastic’s full impact on human health now and in the future.

Reference

Alexander J. Nihart, Marcus A. Garcia, Eliane El Hayek et al, Bioaccumulation of microplastics in decedent human brains, nature medicine, Volume 31 , April 2025, 1114–1119, https://doi.org/10.1038/s41591-024-03453-1

2. R. Marfella, F. Prattichizzo, C. Sardu et al, Microplastics and Nanoplastics in Atheromas and Cardiovascular Events, N Engl J Med 2024;390:900-10. DOI: 10.1056/NEJMoa2309822

3. Micronanoplastics found in artery-clogging plaque in the neck, American Heart Association Meeting Report ,  Scientific Conferences & Meetings, Stroke News & Brain Health, 22 April 2025 available from https://newsroom.heart.org/news/micronanoplastics-found-in-artery-clogging-plaque-in-the-neck

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How MIT’s SLIM Microcrystal Injection Technology is Transforming Long-Acting Treatments for HIV and Contraceptives

by with No Comment Research

Medically Reviewed By Vikas Londhe M.Pharm (Pharmacology)

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Source: Freepik.com

MIT scientists have developed a self-injectable, long-acting drug delivery system using microcrystals. Learn how this innovation could transform contraception and chronic disease treatment.

Imagine a single injection that lasts for months, delivered through a tiny needle you can use yourself, no surgery, no hospital visit. Thanks to a groundbreaking innovation from researchers at Massachusetts Institute of Technology (MIT), that future is now one step closer.

MIT researchers have introduced a groundbreaking innovation that could make this vision a reality. Called SLIM; Self-aggregating Long-acting Injectable Microcrystals this new drug delivery system offers extended release, smaller needle size, and high drug potency, all packed into a single self-administered injection.

A New Way to Take Medicine: Once Every Few Months

Researchers at MIT and Harvard-affiliated Brigham and Women’s Hospital have developed a new drug delivery system called SLIM, short for Self-aggregating Long-acting Injectable Microcrystals.

Instead of needing large needles or surgery, SLIM allows medicine to be injected just under the skin through a tiny needle. Once injected, the medicine automatically forms a small solid “implant” that slowly releases the drug over several months.

This method is especially promising for medications like birth control and HIV medicine where convenience, privacy, and reliability are essential

Global Health Impact

For millions worldwide especially women in under-served regions SLIM could mean:

No need for surgical implants or repeated clinic visits

Empowered self-administration of essential medications

Lower costs and better access to long-term contraception

This new technology is published in the journal Nature Chemical Engineering. The technology also aligns with public health goals from organizations like the WHO and the study is co-funded by Gates Foundation.

How It Works: Tiny Crystals, Big Impact

SLIM works by suspending drug particles called microcrystals in a liquid. When injected into the body, this liquid mixes with the body’s water and causes the particles to clump together into a compact, slow-dissolving depot.

Less pain, smaller needles: Unlike other long-acting injections that require thick, painful needles, SLIM uses fine needles as small as 30 gauge (about the width of a human hair).

Fewer additives: Most long-acting shots rely on lots of added slowly degrading polymers such as polylactic acid or polycaprolactone to slow down drug release. SLIM only needs a tiny amount, reducing risks of side effects and injection pain.

Why It Matters: Especially for Women and Low-Resource Settings

In many parts of the world, women may not have easy access to clinics for repeated contraception or medications. Self-injectable treatments could empower them to take control of their health privately and conveniently.

This is a big step beyond current injectables like Depo-Provera, which lasts 3 months but can’t match the long-lasting protection of surgical implants like Nexplanon. SLIM offers the best of both: long-lasting protection with no surgery required.

A Breakthrough for Contraceptive Delivery

The researchers tested SLIM using levonorgestrel, a common hormonal contraceptive. In lab studies on rats, the SLIM formulation:

In early tests, the SLIM injection worked really well. After being injected, it formed a small, solid implant under the skin that stayed in place. It released the medicine much more slowly up to 5.6 times slower than current options, helping it last longer. The implant stayed strong and stable for over 3 months, and the injection was easy enough to be done at home using a regular small syringe. This means that women could get fewer injections while still having reliable, long-term birth control.

Bottom Line: Why This Matters for the Future of Medicine

The researchers plan to test SLIM with other drugs, especially those that are hard to dissolve in water, like many modern medications.

They’re also studying how to fine-tune drug release and improve safety for human use.

Because of its compact, stable design, SLIM could become a game-changer for treating chronic conditions like mental health, HIV prevention, or cancer with fewer doses and better adherence.

The SLIM system could redefine how we take medicine offering safer, longer-lasting, and more comfortable treatment through self-administered injections. It’s a promising leap toward better access and better outcomes, especially in global health.

Long-acting injectables are a proven way to boost adherence, especially for chronic conditions. But current systems are limited by: Large, painful needles, Viscous, hard-to-inject formulations and Poor suitability for self-administration.

SLIM overcomes these barriers with smart design: high drug loading, low viscosity, and small injection size all while delivering long-lasting effects.

Reference

Feig, V. R., Park, S., Rivano, P. G., et al. (2025). Self-aggregating long-acting injectable microcrystals. Nature Chemical Engineering, 2(3), 209–219. DOI: 10.1038/s44286-025-00194-x

Engineers develop a better way to deliver long-lasting drugs, MIT News, Massachusetts Institute of Technology, 24 March 2025

Owen A, Rannard S. Strengths, weaknesses, opportunities and challenges for long acting injectable therapies: Insights for applications in HIV therapy. Adv Drug Deliv Rev. 2016 Aug 1;103:144-156. Doi: 10.1016/j.addr.2016.02.003. Epub 2016 Feb 23. PMID: 26916628; PMCID: PMC4935562.

McEvoy, Joseph. (2006). Risks versus benefits of different types of long-acting injectable antipsychotics. The Journal of clinical psychiatry. 67 Suppl 5. 15-8.

National Research Council (US) Committee on Population. Contraception and Reproduction: Health Consequences for Women and Children in the Developing World. Washington (DC): National Academies Press (US); 1989. 4, Contraceptive Benefits and Risks.Available from: https://www.ncbi.nlm.nih.gov/books/NBK235069

 

Source of the Video: Giovanni Traverso (YouTube) _Self-Aggregating Long-Acting Injectable Microcystals

Disclaimer: The video is for educational and informational purposes only.

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Novel Oncolytic Virus Therapy Uses Transplant Rejection Pathway to Kill Cancer

by with No Comment Clinical Trails

Medically Reviewed By Vikas Londhe M.Pharm (Pharmacology)

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Newcastle Disease Virus selectively infecting human ovarian cancer cells, visualized with fluorescent markers, highlighting the specificity of the viral targeting, dark background (Source: Freepik.com)

Introduction

In a breakthrough that bring together virology, immunology, and genetic engineering, researcher from State Key laboratory of Targeting Oncology, China have developed a novel oncolytic therapy using a genetically modified Newcastle Disease Virus (NDV) that tricks the immune system into treating cancer cells as if they were foreign organ transplants. This approach, which uses the body’s natural “hyperacute rejection” mechanism typically seen in organ xenotransplants, showed notable results in early clinical trials for patients with advanced, treatment-resistant cancers.

Hyperacute rejection is a type of organ transport rejection that happens within minutes to hours after the transplant. It occurs when the recipient’s immune system already has antibodies that quickly recognize the new organ as foreign and attack it. This rapid immune response causes severe damage to the transplanted organ, often leading to its failure almost immediately.

Xenotransplant is a medical procedure where cells, tissues, or organs are transplanted from one species to another for example, transplanting a pig’s heart into a human.

The Innovation: NDV-GT and the aGal Trigger

The therapy uses a recombinant NDV that expresses the porcaine α1, 3-galactosyltransferase (a1, 3GT) gene, resulting in the presentation of αGal antigens on infected tumor cells. In humans, the αGal antigen is recognized as foreign due to the evolutionary loss of the α1, 3GT gene. Our immune systems naturally produce large amounts of anti-αGal antibodies, primarily from exposure to gut microbiota. This makes the immune system especially reactive to any cell displaying αGal.

By engineering NDV to express this foreign antigen on tumor cells, scientists effectively turned cancer cells into targets of a powerful immune cascade similar to organ rejection. This results in rapid tumor destruction through complement activation, thrombosis, and immune cell infiltration.

Preclinical Success in Monkeys

Using CRISPR-Cas9 technology, the researcher created a realistic liver cancer model in cynomolgus monkeys. The intravenous administration of NDV-GT in these monkeys led to remarkable tumor regression with many tumors disappearing entirely. The therapy not only lysed the cancer cells but also caused thrombotic blockage of tumor blood vessels mimicking hyperacute rejection seen in xenograft failures.

Immune profiling revealed activation of both innate and adaptive immune responses, including CD4+ and CD8+ T cell infiltration, increased expression of granzyme B and perforin, and reduced tumor angiogenesis markers. Importantly, no significant toxicity or organ damage was observed.

Clinical Trial Results

In a phase I interventional clinical trial (ChiCTR2000031980), 20 patients with various advanced, treatment-resistant cancers, including liver, lung, rectal, esophageal, breast, and cervical cancer and melanoma, were treated with intravenous NDV-GT. The results were prominent:

90% Disease Control Rate (DCR): 1 complete response, 6 partial responses, and 11 cases of stable disease.

No severe adverse events: Mild side effects, no cytokine release syndrome, and no detectable viral shedding.

Durable response: Severe patients experienced long-term stabilization o remission lasting up to 36 months.

One standout case involved a patient with advanced hepatocellular carcinoma and lung metastases. After 1.5 months of NDV-GT treatment, most tumors had disappeared, and serum tumor markers dropped significantly. Another patient with metastatic ovarian cancer achieved a partial remission for over a year.

Mechanism of Action

NDV-GT’s effectiveness lies in its dual mechanism :

Direct Oncolysis: NDV naturally prefers and destroys tumor cells while sparing healthy ones.

Immune Amplification: The engineered Gal expression triggers complement-mediated cytotoxicity and T-cell driven immune cascade, simulating a rapid rejection response that targets the tumor as a foreign organ.

This dual action also alleviated the immunosuppressive tumor microenvironment, making tumors more susceptible to further immune attack.

Safety and Implications

Despite the robust immune activation, NDV-GT treatment was well tolerated. No significant increase in neutralizing antibodies or adverse immunogenic effects was observed. The lack of toxicity and the specific targeting of tumor tissue underscore its promise for broader clinical use.

Conclusion

NDV-GT represents a groundbreaking shift in oncolytic virotherapy. Bby mimicking a hyperacute rejection response. This therapy turns the body’s natural defense mechanism against tumors in a way that is both powerful and safe. As phase II-III trials are being prepared, this immune- tricking virus could redefine treatment paradigms for refractory and metastatic cancers.

Reference

1. Liping Zhong, Lu Gan, Bing Wang et al, Hyperacute rejection-engineered oncolytic virus for interventional clinical trial in refractory cancer patients, Cell, Volume 188, Issue 4P1119-1136.E23February 20, 2025.

2. Tricking the Immune System: A New Approach to Targeting Cancer Cells by Mimicking Pig Organs, Insight, 22 Jan 2025, available from https://oncodaily.com/insight/immune-system-229351

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Gold Nanoparticles Show Promise for Vision Restoration, Brown University Study Finds

by with No Comment Research

Medically Reviewed By Vikas Londhe M.Pharm (Pharmacology)

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Image showing gold nanopracticals undergone surface plasmon resonance after exposure to near infra-red light (NIR) Source: Freepik.Com

Researchers at Brown University have developed a groundbreaking vision restoration method that could help people regain vision loss due to retinal diseases like age-related macular degeneration (AMD) and retinitis pigmentosa. These common causes of vision loss damage the retina the light-sensitive layer at the back of the eye leading to progressive blindness. The innovative approach involves using gold nanoparticles, tiny biocompatible particles that can be safely introduced into the eye. When stimulated by near-infrared (NIR) laser light, these nanoparticles activate the remaining retinal cells, allowing them to send visual signals to the brain much like healthy photoreceptors do. This emerging technology has the potential to outperform existing retinal prosthetics by offering a minimally invasive, less painful alternative that could deliver higher-quality vision restoration. With its promise of improved outcomes, enhanced safety, and greater accessibility, this cutting-edge technique could transform the treatment of retinal degeneration and provide hope for millions living with severe vision impairment or blindness.

The Science behind the Innovation

In healthy eyes, specialized cells called photoreceptors rods and cones detect light and convert it into electrical signals. These signals are then passed to other key retinal cells, such as bipolar cells and ganglion cells, which relay the information to the brain, allowing us to perceive images. However, in degenerative eye diseases like age-related macular degeneration (AMD) and retinitis pigmentosa, these critical photoreceptors degenerate and eventually stop functioning, leading to progressive vision loss and, in severe cases, blindness. Remarkably, the inner retinal cells like bipolar and ganglion cells often remain intact and functional even after the photoreceptors are lost. Building on this insight, researchers at Brown University are developing an innovative vision restoration approach that bypasses the damaged photoreceptors entirely. By using advanced technology to directly stimulate the surviving inner retinal cells, this method could restore vision in individuals affected by retinal degeneration. This novel strategy has the potential to revolutionize vision loss treatment by targeting still-viable parts of the eye, offering a powerful alternative to traditional therapies and retinal implants.

Image Source: Brown University from an article: Golden eyes: How gold nanoparticles may one day help to restore people’s vision. Image Showing: How gold nanoparticles work and how it will create visual image in the mind of patient

The research team at Brown University has developed a cutting-edge technique involving the use of gold nanorods ultra-small, rod-shaped particles made of gold delivered directly into the eye through a minimally invasive procedure called intravitreal injection. These gold nanorods are precisely engineered to absorb near-infrared (NIR) light, a safe and effective wavelength that penetrates deep into the eye without causing damage, since it is minimally absorbed by water in the tissue. Once exposed to NIR light, the gold nanorods undergo a process called surface plasmon resonance, which generates localized and highly controlled heat. This subtle thermal energy can activate surrounding retinal nerve cells in two key ways: by inducing tiny electric currents across their membranes or by triggering specialized ion channels that respond to changes in temperature. This innovative approach offers a promising new method for restoring vision by reactivating the surviving retinal cells in individuals affected by degenerative eye diseases such as macular degeneration and retinitis pigmentosa. It also holds the potential to be a safer, more precise alternative to traditional retinal implants or surgical treatments.

Promising Results in Preclinical Studies

In experimental tests with mice suffering from retinal damage, the research team injected gold nanoparticles into the animals’ eyes and projected patterned near-infrared (NIR) laser light onto the retina. These light patterns, shaped like simple images, were designed to test whether the NIR light could stimulate the remaining healthy retinal cells. Using a powerful technique called calcium imaging; the scientists observed that the bipolar and ganglion cells key players in the visual signalling pathway responded to the laser patterns in a manner similar to how they would react to natural light in a healthy retina. Additionally, brain imaging revealed increased activity in the visual cortex, the region of the brain responsible for processing visual information. This indicated that the brain was once again receiving visual input, a strong sign that sight could potentially be restored. Even more promising, the gold nanoparticles remained stable and safely positioned within the eye for several months, with no signs of toxicity or inflammation. These results highlight the long-term potential of this non-invasive, laser-activated nanoparticle therapy as a safe and effective treatment for restoring vision in people affected by retinal degenerative diseases.

Advantages over Traditional Retinal Prosthetics

This new nanoparticle based method has several key benefits compared to current retinal prosthetic devices. First, it is minimally invasive unlike traditional devices that require complex surgery to implant electrodes; this approach only needs a simple injection into the eye, a common and low-risk procedure in eye care. Second, it offers much higher resolution. Existing devices are limited because they can only stimulate certain fixed spots in the retina, often resulting in blurry or narrow vision. In contrast, the nanoparticles can be targeted more precisely, even down to individual cells, and their stimulation pattern can be changed as needed, potentially restoring a full, detailed field of vision. Lastly, because it uses near-infrared (NIR) light, which doesn’t affect visible light, this method can work alongside any natural vision a person still has, making it a more flexible and user-friendly solution.

Future Outlook

Researchers aim to transform this groundbreaking vision restoration technology into a wearable device, such as smart glasses or high-tech goggles equipped with a camera and a near-infrared (NIR) laser system. The camera would continuously capture visual input from the surrounding environment, while the NIR laser projects precise light patterns onto the retina. These patterns would activate specially designed gold nanoparticles, allowing the user to perceive visual images without the need for invasive procedures. Although more research is needed to confirm long-term safety and effectiveness in larger animal models and eventually in humans, this innovative approach could revolutionize treatment options for millions suffering from retinal diseases and vision loss. By eliminating the need for genetic modification or complex surgical implants, this technology offers a potentially safer, more accessible and cost-effective alternative to current retinal prosthetics, paving the way for broader adoption and improved quality of life for patients worldwide.

Reference:

1. 1.Jiarui Nie, Kyungsik Eom, Hafithe M. AlGhosain, ntravitreally Injected Plasmonic Nanorods Activate Bipolar Cells with Patterned Near-Infrared Laser Projection ACS Nano 2025, 19, 12, 11823–11840, https://doi.org/10.1021/acsnano.4c14061

2. Golden eyes: How gold nanoparticles may one day help to restore people’s vision, 16 April 2025, Brown University.

3. Chen F , Si P , de la Zerda A , Jokerst JV , Myung D . Gold nanoparticles to enhance ophthalmic imaging. Biomater Sci. 2021 Jan 21;9(2):367-390. doi: 10.1039/d0bm01063d. Epub 2020 Oct 15. PMID: 33057463; PMCID: PMC8063223.

4. Natarajan S. Retinitis pigmentosa: a brief overview. Indian J Ophthalmol. 2011 Sep-Oct; 59(5):343-6. Doi: 10.4103/0301-4738.83608. PMID: 21836337; PMCID: PMC3159313.

5. Giuliana Gagliardi, Karim Ben M’Barek, Olivier Goureau, Photoreceptor cell replacement in macular degeneration and retinitis pigmentosa: A pluripotent stem cell-based approach, Progress in Retinal and Eye Research, Volume 71, 2019, Pages 1-25, https://doi.org/10.1016/j.preteyeres.2019.03.001

6. Treating retinal disease with tiny gold particles, 24 April 2025, Association of optometrist, available from https://www.aop.org.uk/ot/news/2025/04/24/treating-retinal-disease-with-tiny-gold-particles

7. NEI-funded researchers test new visual prosthesis system to restore vision, 18 April 2025, National Eye Institute, available from https://www.nei.nih.gov/about/news-and-events/news/nei-funded-researchers-test-new-visual-prosthesis-system-restore-vision