The Food and Drug Administration (FDA) has accepted the biologics license application (BLA) and granted priority review designation for pegunigalsidase alfa (PRX–102; Protalix BioTherapeutics, Karmiel, Israel) for the proposed treatment of individuals with Fabry disease. The BLA was submitted via the FDA's accelerated approval pathway. Pegunigalsidase alfa is purposefully designed, long-acting recombinant, PEGylated, cross-linked α-galactosidase-A investigational product candidate.
The BLA submission includes a comprehensive set of preclinical, clinical, and manufacturing data compiled from the completed phase 1/2 clinical trial of pegunigalsidase alfa. This includes
the related extension study, interim clinical data from the phase 3 BRIDGE (NCT03018730) switch-over study, and safety data from ongoing clinical studies of pegunigalsidase alfa in participants 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."
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 6 months compared with 10 months under standard review. Pegunigalsidase alfa was granted fast track designation by the FDA in January 2018. 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.
Cyrus A. Raji, MD, PhD; Somayeh Meysami, MD; and Mario F. Mendez, MD, PhD
Adam M. Staffaroni, PhD; Elena Tsoy, PhD; Jack Taylor, BS; Adam L. Boxer, MD, PhD; and Katherine L. Possin, PhD
Omar Bushara, BA; Rimas V. Lukas, MD; and Jessica W. Templer, MD