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Positive CHMP opinion for pegunigalsidase alfa for the treatment of Fabry disease. - Chiesi Global Rare Diseases + Protalix BioTherapeutics, Inc.

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Published:26th Feb 2023

Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people affected by rare diseases, and Protalix BioTherapeutics, Inc. a biopharmaceutical company, announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) 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 alpha-GalactosidaseA (alpha 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 III BALANCE, BRIDGE, and BRIGHT clinical trials, the Phase 1/II 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.

Condition: Fabry Disease
Type: drug

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