The Company, together with Chiesi, met with the FDA on October 15 to discuss key information on pegunigalsidase alfa to be included in a proposed BLA filing under the Accelerated Approval pathway. The Company and Chiesi report that they have reached alignment with the FDA on the Accelerated Approval pathway for pegunigalsidase alfa. The BLA, as expected to be submitted to the FDA, will include data from the Company's completed phase I/II clinical trials of pegunigalsidase alfa and from its ongoing phase III BRIDGE clinical trial.
"The confirmation by the FDA regarding the proposed BLA submission, together with the finalization of the BALANCE study enrollment (78 patients) and the very promising interim results from the BRIDGE study, are three recent significant consecutive milestones achieved with respect to our Fabry late-stage clinical program," said Dror Bashan, Protalix's President and Chief Executive Officer. "We are committed to the completion of this program with positive results for the benefit of the Fabry patient community."
The Company and Chiesi also announced that they have reached an agreement with the FDA regarding the ongoing BALANCE study, as currently designed, serving as the confirmatory trial for PRX‑102. A confirmatory trial is required to convert a BLA approved under Accelerated Approval into a traditional approval.
The Company and Chiesi expect to submit the BLA to the FDA by April of 2020. The FDA indicated that the nonclinical data, the clinical data, the safety database and manufacturing data are sufficient to support the BLA submission, and that no additional clinical trials will be required for the proposed BLA submission. The Company and Chiesi expect that the fully electronic BLA submission will include a comprehensive set of preclinical, clinical and manufacturing related information on pegunigalsidase alfa. If approved, the Company will be eligible to receive a milestone payment from Chiesi.
"We have now had three successful interactions with the FDA during 2019, which built on our long-term relationship with the Agency and made our regulatory path forward for pegunigalsidase alfa clear," said Dr. Einat Almon, Protalix's Senior Vice President, Product Development. "We expect that alignment with the FDA on the Accelerated Approval pathway for pegunigalsidase alfa results in our being significantly closer to bringing an approved product to market to help Fabry patients. Together with our partner Chiesi, we look forward to completing the application process, as well as continuing with our double blind head-to-head phase III BALANCE clinical trial, which we feel will further strengthen the position of pegunigalsidase alfa within the Fabry patient community."