Written By: Pharmacally Medical News Desk
The EMA CHMP, at its November 2025 meeting, recommended ten new medicines for approval across a spectrum of therapeutic areas.
Dawnzera (donidalorsen)
Dawnzera (donidalorsen), developed by Otsuka, received a positive recommendation for the prevention of recurrent hereditary angioedema (HAE) in patients aged 12 and older. This RNA-targeted therapy inhibits plasma prekallikrein synthesis, reducing bradykinin production that triggers HAE attacks. The pivotal Phase 3 OASIS-HAE trial showed an impressive 81% reduction in monthly HAE attacks compared to placebo over 24 weeks, demonstrating substantial clinical benefit.
GalenVita
GalenVita, based on Gallium-68 chloride (Ge-68/Ga-68), serves as a radionuclide generator for PET imaging in oncology, improving tumor visualization via radiolabeled tracers. Its mechanism involves providing Ga-68, a positron-emitting radioisotope used in diagnostic imaging. While clinical trials focus on its radiolabeling efficiency, it supports enhanced diagnostic precision in cancer management.
Inluriyo (imlunestrant)
Inluriyo (imlunestrant), by Eli Lilly Nederland B.V., is indicated for advanced or metastatic estrogen receptor-positive (ER+), HER2-negative breast cancer patients with ESR1 mutations. This oral selective estrogen receptor degrader blocks ESR1 signaling to inhibit tumor growth. The EMBER-3 Phase 3 trial reported a median progression-free survival of 5.5 months versus 3.8 months with standard endocrine therapy, reflecting significant efficacy (HR=0.62, p=0.0008).
Teizeild (teplizumab)
Teizeild (teplizumab), from Sanofi Winthrop, is recommended to delay progression to stage 3 type 1 diabetes in individuals aged eight or older with stage 2 disease. This anti-CD3 monoclonal antibody modulates T-cell activity to preserve insulin-producing beta cells. The TrialNet study demonstrated that teplizumab extended the median time to clinical diabetes diagnosis from 27.1 to 59.6 months (HR=0.41, p=0.006), highlighting its disease-modifying potential.
Vacpertagen
Vacpertagen is an acellular pertussis vaccine designed for the prevention of whooping cough across all age groups. It uses genetically inactivated pertussis toxin and filamentous hemagglutinin components to elicit strong immunity. Phase 3 trials showed robust antibody responses with superior seroconversion rates compared to traditional vaccines, addressing concerns of waning immunity.
Waskyra (etuvetidigene autotemcel)
Waskyra (etuvetidigene autotemcel), developed by Fondazione Telethon, is an autologous gene therapy targeting Wiskott-Aldrich syndrome in patients lacking suitable stem cell donors. The therapy involves ex vivo correction of hematopoietic stem cells to restore immune function. The multinational pivotal trial revealed marked reductions in severe infections and bleeding episodes, with improved immune parameters, signifying a breakthrough in rare genetic immune disorders.
Enzalutamide
Enzalutamide Accordpharma, an androgen receptor inhibitor by Accord Healthcare, targets prostate cancer by blocking androgen-mediated tumor proliferation. In the Phase 3 ARCHES trial, enzalutamide reduced mortality risk by 30% and significantly extended progression-free and overall survival, underscoring its role in advanced prostate cancer therapy.
Ondibta (insulin glargine)
Ondibta (insulin glargine), from Gan & Lee Pharmaceuticals, is a long-acting basal insulin analog for managing type 1 and type 2 diabetes mellitus. Biosimilarity trials comparing Ondibta to the reference product Lantus demonstrated equivalent safety and efficacy profiles, ensuring reliable glycemic control for patients requiring insulin therapy.
Osqay
Osqay, a denosumab biosimilar, indicated for osteoporosis and bone loss disorders, works by inhibiting the receptor activator of nuclear factor kappa-B ligand (RANKL), thereby reducing bone resorption. Clinical biosimilarity studies met all efficacy and safety endpoints, providing an alternative to the reference biologic with comparable therapeutic outcomes.
Teduglutide
Teduglutide Viatris, a glucagon-like peptide-2 (GLP-2) analog from Viatris Limited, is indicated for short bowel syndrome to improve nutrient absorption. Clinical trials, including the STEPS program, demonstrated increased intestinal absorption and reduced dependence on parenteral nutrition, improving patient quality of life.
These recommendations represent important advances across diverse fields including rare diseases, oncology, endocrinology, infectious diseases, and gene therapy, highlighting EMA’s commitment to facilitating access to innovative and effective therapies in the European Union.
References
Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 10-13 November 2025, 14 November 2025, European Medicine Agency, https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-10-13-november-2025