FDA issues a Complete Response Letter relating to the BLA for PRX 102 for the proposed treatment of Fabry disease.- Protalix BioTherapeutics,

Protalix BioTherapeutics, Inc. provided an update regarding the Complete Response Letter (CRL) received from the FDA regarding the Biologics License Application (BLA) seeking accelerated approval of pegunigalsidase alfa (PRX 102) for the proposed treatment of adult patients with Fabry disease. In the CRL, the FDA noted that an inspection of Protalix's manufacturing facility in Carmiel, Israel, including the FDA's subsequent assessment of any related findings, is required before the FDA can approve the BLA. Due to travel restrictions, the FDA was unable to conduct the required inspection during the review cycle. The FDA explained that it will continue to monitor the public health situation as well as travel restrictions, and is actively working to define an approach for scheduling outstanding inspections. With respect to the third-party facility in Europe at which fill and finish processes are performed for PRX 102, due to COVID–19, the FDA reviewed records under Section 704(a)(4) of the Federal Food, Drug, and Cosmetic Act in lieu of a pre-licensing inspection. In the CRL, the FDA stated that it will communicate remaining issues to the facility in order to seek prompt resolution of any pending items. In addition to the foregoing, the FDA noted that Fabrazyme was recently converted to full approval which must be addressed in the context of any potential resubmission seeking accelerated approval of PRX 102. Protalix intends to work collaboratively with the agency to identify the most expeditious pathway to approval, including accelerated approval. Protalix remains confident that it will be able to work with the FDA to resolve these issues and provide a new, alternative drug to Fabry patients. Protalix intends to request a Type-A meeting with the FDA, and will provide further updates on next steps after the meeting.