Protalix and Chiesi Secure EU Approval for 2mg/kg Every-Four-Weeks Dose, Triggering $25M Payment
Protalix and Chiesi secured European Commission approval for a 2 mg/kg every-four-weeks pegunigalsidase alfa regimen in adults stable on enzyme replacement therapy for Fabry disease, reducing infusion frequency from biweekly. The approval triggered a $25 million regulatory milestone payment and may lower treatment burden for patients.
1. EU Approval Secures New Dosing Regimen
The European Commission approved a 2 mg/kg every-four-weeks dosing schedule for pegunigalsidase alfa in adults with stable enzyme replacement therapy for Fabry disease, extending infusion intervals from every two weeks to every four weeks and improving treatment convenience.
2. Study Data Underpinning Approval
This decision was based on outcomes from the 52-week open-label BRIGHT study and its ongoing extension, which demonstrated that the extended-interval regimen maintained safety, efficacy and pharmacokinetic profiles comparable to the biweekly schedule.
3. Financial and Market Implications
The approval triggered a $25 million regulatory milestone payment from Chiesi Global Rare Diseases, enhancing Protalix’s cash position and setting the stage for broader market access of pegunigalsidase alfa across European Union healthcare systems.