FDA’s Decision on Recessive Dystrophic Epidermolysis Bullosa Treatment Delayed

The Food and Drug Administration (FDA) has issued a Complete Response Letter to Abeona Therapeutics regarding the Biologics License Application for prademagene zamikeracel for the treatment of recessive dystrophic epidermolysis bullosa, a rare connective tissue disorder.

Recessive dystrophic epidermolysis bullosa (RDEB) is characterized by extensive blistering and severe skin wounds caused by a mutation in the COL7A1 gene. Prademagene zamikeracel (pz-cel) is an autologous cell therapy that consists of epidermal sheets that deliver functional COL7A1 genes into the patient’s own skin cells to enable normal type VII collagen expression and facilitate wound healing.

In the letter, the FDA requested the Company provide additional information to satisfy Chemistry Manufacturing and Controls (CMC) requirements before a decision on approval could be made. The deficiencies noted in the CRL were not related to efficacy or safety data. No additional clinical trials were required to support the approval.

The BLA was supported by data from the phase 3 VIITAL study (ClinicalTrials.gov Identifier: NCT04227106), which evaluated the safety and efficacy of pz-cel in the treatment of large, chronic RDEB wounds. The trial included 43 large chronic wound pairs in 11 patients with RDEB. Findings showed 81.4% of pz-cel-treated wounds achieved 50% or greater wound healing compared with 16.3% of untreated control wounds (P <.001). Additionally, a statistically significant improvement in pain reduction associated with wound dressing changes was observed with pz-cel when compared with untreated control wounds (P =.0002).

“While we are surprised and disappointed by this CRL, we are committed to providing the CMC information necessary to respond to the agency’s asks, with the goal of bringing pz-cel to patients with RDEB as quickly as possible,” said Vish Seshadri, CEO  of Abeona. “We are already hard at work generating the additional CMC information, and we expect that all of FDA’s requests will be addressable in a reasonable timeframe. We anticipate completing the BLA resubmission in the third quarter of 2024 with necessary updates to fully satisfy all the deficiencies outlined in the CRL.”