The European Medicines Agency (EMA) has validated a variation submission for pegunigalsidase alfa (Elfabrio), seeking approval for a less frequent dosing regimen in adult patients with Fabry disease. The proposed regimen involves administering 2 mg/kg of the drug every four weeks, a change from the currently approved 1 mg/kg dose given every two weeks. This development aims to reduce the treatment burden for patients while maintaining efficacy.
The application is supported by data from the completed Phase 3 PB-102-F50 (BRIGHT) trial and its ongoing extension study CLI-06657AA1-03. These studies evaluated the safety and efficacy of the 2 mg/kg every four weeks dosing regimen in adult Fabry disease patients previously treated with agalsidase-alfa or -beta every two weeks. The data included a revised Population-PK model and new exposure-response analyses.
Giacomo Chiesi, Executive Vice President of Chiesi Global Rare Diseases, stated, "The validation of this variation application is an important milestone in our efforts to reduce the burden of treatment for some adult patients living with Fabry disease who continue to experience unmet medical needs. We are committed to delivering innovative therapies and solutions for people living with Fabry disease, their families and caregivers."
Dror Bashan, Protalix's President and Chief Executive Officer, added, "Based on study results, we believe in the potential of pegunigalsidase alfa 2 mg/kg administered every four weeks to be a beneficial, alternative dosing option for some adults living with Fabry disease. Together with Chiesi, we remain committed to meeting the needs of people with Fabry disease and bringing additional therapeutic options to market. We look forward to continuing to work closely with the agency in the months ahead."
Elfabrio's Safety Profile
Elfabrio carries a warning for hypersensitivity reactions, including anaphylaxis. In clinical trials, 14% of Elfabrio-treated patients experienced hypersensitivity reactions, and 3% experienced anaphylaxis. Common adverse reactions (≥15%) included infusion-associated reactions, nasopharyngitis, headache, diarrhea, fatigue, nausea, back pain, pain in extremity, and sinusitis.
About Fabry Disease
Fabry disease is a rare, inherited lysosomal storage disorder caused by a deficiency in the enzyme alpha-galactosidase A, leading to the accumulation of globotriaosylceramide (Gb3) in various cells throughout the body. This accumulation can cause a range of symptoms, including pain, kidney disease, heart disease, and stroke. Enzyme replacement therapy aims to address the underlying enzyme deficiency and reduce Gb3 accumulation.
Companies Involved
Chiesi Global Rare Diseases, a business unit of the Chiesi Group, focuses on delivering therapies for rare diseases. Protalix BioTherapeutics, Inc. develops and commercializes recombinant therapeutic proteins using its ProCellEx plant cell-based expression system. The two companies have partnered for the global development and commercialization of pegunigalsidase alfa.
