Research Update: Pz-cel for RDEB
Back in November 2023, the U.S. Food and Drug Administration granted priority review for Pz-cel (prademagene zamikeracel) and set a PDUFA date of May 25, 2024, which is when they would have decided on whether the treatment is approved or not.
However, earlier this week, Abeona Therapeutics received a Complete Response Letter (CRL) from the FDA stating that they are unable to approve the application in its current form. The FDA is requiring Abeona to provide more information about how the treatment is made and tested, per their Chemistry Manufacturing and Controls (CMC) requirements.
Notably and most importantly, the FDA did not find any issues or have any questions about the clinical data, aka how well it worked, or the safety of pz-cel, and they did not ask for more clinical data or additional trials. The Abeona team is confident they can address the FDA’s concerns and hope to complete the Biologics License Application (BLA) resubmission by the third quarter of this year.
"Clearly this is a setback for those with DEB who are waiting with bated breath to use another advanced therapeutic product aimed at easing the burdens EB presents,” said Brett Kopelan, MA, Executive Director of debra of America. “With that said, of the 12 times the FDA has issued a CRL for CMC issues, 11 have been resolved and the therapeutic was approved. So, I really like our chances and am cautiously optimistic that the FDA will approve pz-cel in the near future. I have all the confidence in the world that Abeona will successfully provide the additional data and processes that the FDA has requested.”
Pz-cel is Abeona’s investigational autologous, COL7A1 gene-corrected epidermal sheets for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB). It has demonstrated promising results in their Phase 3 VIITAL™ and Phase 1/2a trials, showing that the application of pz-cel once on large and chronic wounds helped them heal and reduced pain.
For more information, read the Abeona Therapeutics press release.
