- In November 2024, Abeona Therapeutics Inc. announced the FDA’s acceptance of the resubmission of its Biologics License Application (BLA) for prademagene zamikeracel (pz-cel), a novel autologous cell-based gene therapy. This investigational therapy aims to treat recessive dystrophic epidermolysis bullosa. The FDA set April 29, 2025, as the target date for a decision under the Prescription Drug User Fee Act (PDUFA)
- In May 2023, Krystal Biotech, Inc. revealed that the U.S. FDA had approved VYJUVEK (beremagene geperpavec-svdt) for treating dystrophic epidermolysis bullosa in individuals aged six months and older. VYJUVEK targets the genetic cause of the disease by delivering functional copies of the COL7A1 gene, promoting wound healing, and enabling long-term COL7 protein expression with redosing
