The Food and Drug Administration (FDA) has accepted the resubmitted Biologics License Application (BLA) for pegunigalsidase alfa for the treatment of adults with Fabry disease.

Fabry disease is a rare genetic progressive disorder caused by deficient activity of the lysosomal α-galactosidase-A enzyme, which leads to progressive buildup of globotriaosylceramide (Gb3) in blood vessel walls throughout the body. Pegunigalsidase alfa is a long-acting recombinant, PEGylated, cross-linked α-galactosidase-A investigational product candidate. 

The BLA was originally accepted and granted Priority Review in August 2020 based on a phase 1/2 clinical trial, which included the related extension study, interim clinical data from the phase 3 BRIDGE switch-over study and safety data.

The FDA subsequently issued a Complete Response Letter requesting an inspection of Protalix’s manufacturing facility in Carmiel, Israel. The letter did not report any concerns related to the safety and efficacy of pegunigalsidase alfa.

In order to support a traditional approval, the Company resubmitted the BLA with a comprehensive set of clinical and manufacturing data. The application now includes data from three phase 3 trials: BALANCE, BRIDGE, and BRIGHT (ClinicalTrials.gov Identifier: NCT02795676, NCT03018730, NCT03180840, respectively), along with the phase 1/2 trial.

A Prescription Drug User Fee Act action date of May 9, 2023 has been set for the application.

Reference

Protalix BioTherapeutics and Chiesi Global Rare Diseases announce US Food and Drug Administration acceptance of a resubmitted Biologics License Application for pegunigalsidase alfa for the proposed treatment of Fabry disease. News release. Protalix BioTherapeutics and Chiesi Global Rare Diseases. Accessed December 5, 2022. https://www.prnewswire.com/news-releases/protalix-biotherapeutics-and-chiesi-global-rare-diseases-announce-us-food-and-drug-administration-acceptance-of-a-resubmitted-biologics-license-application-for-pegunigalsidase-alfa-for-the-proposed-treatment-of-fabry-disease-301694436.html.