European Commission approves every-4-week (2 mg/kg) dosing of Elfabrio (pegunigalsidase alfa) for adults with Fabry disease stable on enzyme replacement therapy.
Written By: Karthik Teja Macharla, PharmD
Reviewed By: Pharmacally Editorial Team
The European Commission (EC) has approved a new dosing regimen for Elfabrio® (pegunigalsidase alfa), allowing administration of 2 mg/kg once every four weeks (E4W) in adults with Fabry disease who are stable on enzyme replacement therapy (ERT). The approval expands treatment flexibility for eligible patients across the European Union.
The decision follows a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) and marks an important update to the treatment’s dosing schedule.
Pegunigalsidase alfa is developed by Protalix BioTherapeutics and commercialized in partnership with Chiesi Global Rare Diseases, the rare disease division of the Chiesi Group.
Giacomo Chiesi, Executive Vice President of Chiesi Global Rare Diseases, said the approval offers a meaningful advance for patients and families managing the lifelong burden of Fabry disease. He noted that extending the infusion interval from every two weeks to every four weeks could help reduce treatment disruption and provide greater flexibility in daily life.
Clinical expert Prof. Aleš Linhart, emphasized that treatment decisions in Fabry disease must balance effective disease control with the realities of lifelong therapy. According to Linhart, the newly approved dosing option may help reduce the cumulative treatment burden for appropriate patients while maintaining continuity of care.
Dror Bashan, President and Chief Executive Officer of Protalix BioTherapeutics, said the authorization strengthens the treatment landscape for Fabry disease in the European Union. He added that the additional dosing approach reflects progress in optimizing long-term care while supporting both patients and healthcare systems.
Patient advocacy groups also welcomed the approval. Mary Pavlou, President of the Fabry International Network (FIN), said that reducing the frequency of infusion visits could ease the ongoing treatment burden for many patients and families living with the condition.
The EC decision was supported by data from the BRIGHT study (NCT03180840) (PB-102-F50), an open-label switch-over trial evaluating the safety profile, efficacy, and pharmacokinetics of the 2 mg/kg every-four-weeks regimen over 52 weeks. The findings are further supported by an ongoing open-label extension study (NCT03614234).
Under the companies’ collaboration agreement, Protalix BioTherapeutics will receive a $25 million regulatory milestone payment from Chiesi following the EC approval of the new dosing regimen.
Fabry disease is a rare inherited lysosomal storage disorder caused by mutations in the GLA gene, resulting in deficiency of the enzyme alpha-galactosidase A. The enzyme deficiency leads to the accumulation of globotriaosylceramide (GL-3) in cells throughout the body, affecting multiple organs including the heart, kidneys, nervous system, and skin.
The disease can cause a wide range of symptoms such as chronic pain, fatigue, gastrointestinal problems, reduced ability to sweat, progressive kidney dysfunction, heart complications, and an increased risk of stroke. Fabry disease affects both males and females and can appear from childhood through adulthood, often with delayed diagnosis.
Early detection and timely access to treatments such as enzyme replacement therapy or pharmacological chaperone therapy are critical to managing symptoms and slowing disease progression.
Reference
Chiesi Global Rare Diseases and Protalix BioTherapeutics Announce European Commission Approval of Additional Dosing Regimen of Every Four Weeks for Elfabrio® (pegunigalsidase alfa), 09 March 2026, chiesirarediseases.com/media/20260309-chiesi-global-rare-diseases-and-protalix-biotherapeutics-announce-european-commission-approval-of-additional-dosing-reg
Safety, Efficacy, & PK of PRX-102 in Patients with Fabry Disease Administered Intravenously Every 4 Weeks (BRIGHT), ClinicalTrials.gov ID NCT03180840, https://clinicaltrials.gov/study/NCT03180840
Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients, ClinicalTrials.gov ID NCT03614234, https://clinicaltrials.gov/study/NCT03614234
About the Writer
Karthik Teja Macharla, PharmD,
He is a Pharm.D. graduate with a strong interest in clinical research, pharmacovigilance, and medical writing. In his words, he is passionate about converting complex medical information into clear, evidence-based scientific communication, committed to contributing to patient safety and advancing healthcare through accurate and impactful medical content.