FDA Accepts Resubmitted BLA for Fabry Disease

A Prescription Drug User Fee Act action date for pegunigalsidase alfa is set for May 9, 2023.

The FDA has accepted the resubmitted biologics license application for pegunigalsidase alfa to treat adult patients with Fabry disease. Fabry disease is a life-threatening, rare genetic disorder that can cause pain and impaired peripheral sensation and can lead to organ failure, particularly of the kidneys. It is caused by a deficiency of the lysosomal α–Galactosidase–A enzyme, which leads to abnormal deposits of a fatty substance called globotriaosylceramide (Gb3) in blood vessel walls. Fabry disease occurs in one person per 40,000 to 60,000.

Pegunigalsidase alfa (PRX–102) is an enzyme replacement therapy that was developed by Protalix BioTherapeutics and Chiesi Global Rare Diseases. It is a long-acting and chemically modified stabilized version of the recombinant α–Galactosidase–A enzyme. A Prescription Drug User Fee Act (PDUFA) action date is set for May 9, 2023.

The BLA resubmission includes a comprehensive set of clinical and manufacturing data. The data were compiled from studies that involved more than 140 Fabry disease patients with up to five years of follow up, including all three completed studies in the PRX-102 phase 3 clinical program including the BALANCE study, the BRIDGE study and the BRIGHT study, as well as the phase 1/2 clinical trial of PRX–102. The phase 1/2 data includes data compiled from the related extension study succeeding the phase 1/2 study. The BLA resubmission also includes safety data compiled from the ongoing phase 3 extension studies of PRX–102.

Related: Protalix, Chiesi Resubmit BLA to Treat Fabry Disease

The BLA has been resubmitted last month after receiving complete response letter from the FDA in April 2021. At the time, the agency indicated that travel restrictions limited the ability to complete inspections of Protalix’s manufacturing facility in Israel.