Protalix BioTherapeutics Announces FDA Acceptance Of BLA For The Treatment Of Fabry Disease And Grants Priority Review

CARMIEL, Israel, Aug. 11, 2020 -- Protalix BioTherapeutics, Inc. (NYSE American: PLX) (TASE: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell-based protein expression system, or the Company, together with its development and commercialization partner Chiesi Global Rare Diseases, a unit of Chiesi, an international research-focused healthcare group, today announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) and granted Priority Review designation for pegunigalsidase alfa for the proposed treatment of adult patients with Fabry disease. The BLA was submitted via the FDA's Accelerated Approval pathway. Pegunigalsidase alfa is the Company's purposefully designed, long-acting recombinant, PEGylated, cross-linked α-galactosidase-A investigational product candidate. The FDA set an action date of January 27, 2021, under the Prescription Drug User Fee Act (PDUFA). The FDA also indicated in the BLA filing communication letter that it is not currently planning to hold an advisory committee meeting to discuss the application.

Protalix Biotherapeutics Logo (PRNewsfoto/Protalix Biotherapeutics Inc)

Priority Review is granted to therapies that the FDA determines have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions. This designation shortens the FDA review period following the acceptance of the BLA to six months compared to 10 months under standard review. Pegunigalsidase alfa was granted Fast Track designation by the FDA in January 2018.

The BLA submission includes a comprehensive set of preclinical, clinical and manufacturing data compiled from the Company's completed Phase I/II clinical trial of pegunigalsidase alfa, including the related extension study succeeding the Phase I/II clinical trial, interim clinical data from the Phase III BRIDGE switch-over study and safety data from the Company's on-going clinical studies of PRX–102 in patients receiving 1 mg/kg every other week.

"The FDA's acceptance of the BLA and grant of priority review for PRX-102 are significant achievements for Protalix and Chiesi, and represent a crucial step forward as we look to establish a new treatment option to the Fabry patient community," said Dror Bashan, Protalix's President and Chief Executive Officer. "Based on the encouraging results for PRX-102 we have seen to date, we are eager to continue discussions with the FDA and to continue our other development efforts for PRX-102, as marketing approval of PRX-102 is our top priority."

"PRX-102 represents an important advance in research with the potential to deliver significant advantages to patients with Fabry disease," said Giacomo Chiesi, Head of Global Rare Diseases. "We are very encouraged by the strong interest in this therapy among both patients and clinicians and we look forward to the prospect of making it available to patients around the world who can benefit from treatment."

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