Chiesi Global Rare Diseases and Protalix BioTherapeutics, Inc. announced that the European Medicines Agency's Committee for Medicinal Products for Human Use adopted a positive opinion, recommending marketing authorization for PRX–102 (pegunigalsidase alfa), the first and only pegylated enzyme for the treatment of adult patients with Fabry disease. PRX–102 is a novel recombinant human a–Galactosidase–A (a–Gal–A) enzyme being investigated as an enzyme replacement therapy (ERT) for the treatment of Fabry disease. The positive CHMP opinion was based on a marketing authorization application (MAA) that includes positive data from a comprehensive set of preclinical, clinical and manufacturing studies evaluating PRX–102.

The clinical development program includes the completed Phase 3 BALANCE, BRIDGE, and BRIGHT clinical trials, the Phase 1/2 clinical trial, and ongoing related extension studies that combined represent more than 400 years of exposure to PRX–102. PRX–102 has been studied in more than 140 patients, consisting of both ERT–naïve and ERT–experienced patients, and includes a head–to–head trial versus agalsidase beta. The CHMP opinion is now referred for final action to the European Commission (EC).

A final EC decision on the MAA is expected in the beginning of May 2023.