FDA denies Abeona’s recessive dystrophic epidermolysis bullosa treatment
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Key takeaways:
- The FDA issued a complete response letter based on lack of information on certain manufacturing and release testing methods.
- Abeona plans to resubmit its application in the third quarter of 2024.
The FDA did not approve Abeona Therapeutics’ biologics license application for prademagene zamikeracel for recessive dystrophic epidermolysis bullosa, the company announced in a press release.
During a late cycle review meeting in March as well as in a subsequent information request, the FDA informed Abeona Therapeutics that certain additional data were needed to satisfy chemistry manufacturing and controls (CMC) requirements before the application for prademagene zamikeracel (pz-cel) for recessive dystrophic epidermolysis bullosa (RDEB) could be approved.
After submitting a plan to provide the CMC data, the FDA issued a complete response letter (CRL) stating that the proposed timing of the data submission would not allow sufficient time to complete its review by the PDUFA date on May 25.
“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,” Vishwas Seshadri, MBA, CEO of Abeona, said in the release.
According to the press release, the additional data needed to fulfill the CMC requirements include information on certain manufacturing and release testing methods. The company has pointed out that the FDA did not identify any problems related to pz-cel’s efficacy or safety.
Abeona Therapeutics shares fell 53% just hours after its application for pz-cel was denied approval. Nevertheless, the company remains optimistic about the future of pz-cel for RDEB.
“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,” Seshadri said.
Reference:
- Abeona Therapeutics shares down 53% after FDA complete response letter. https://www.marketwatch.com/amp/story/abeona-therapeutics-shares-down-53-after-fda-complete-response-letter-23d0d3c8. Published April 22, 2024. Accessed April 23, 2024.
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