Abeona Therapeutics’ shares dropped by almost 50% pre-market Tuesday after receiving an FDA complete response letter for its cell therapy for epidermolysis bullosa. The asset’s PDUFA date is also delayed from the original May 25 date.
After a meeting with Abeona in March, the FDA noted more information on chemistry, manufacturing and control was needed before its lead candidate, dubbed prademagene zamikeracel (pz-cel), can be approved in the rare skin condition. The FDA requested further data on manufacturing and release testing methods, including observations found during the agency’s pre-license inspection.
The company is seeking approval for pz-cel in patients with recessive dystrophic epidermolysis bullosa. Abeona shares $ABEO plummeted by as much as 48% pre-market after a Monday afternoon press release.
Abeona said it will submit its biologics license application (BLA) with further data and a full validation report in the third quarter of 2024, which means the FDA will not be able to make a decision by May 25. Abeona has discussed its plans with the FDA in a second informal meeting, the company said.
In June 2023, pz-cel’s BLA submission was delayed after the FDA requested for more manufacturing information, such as retroviral vector comparability data between internal and external production. The BLA for pz-cel was submitted in November 2023 and granted priority review by the FDA.
Last month, Abeona was handed a Form 483 related to process control issues for pz-cel, after an inspection of its site in Cleveland, OH.
Abeona said the FDA found no deficiencies in the cell therapy’s efficacy or safety profiles and that no additional clinical trials are needed.