FDA Issues Complete Response Letter to Abeona Therapeutics Regarding PZ-Cel for Recessive Dystrophic Epidermolysis Bullosa
Citing a need for more Chemistry Manufacturing and Controls information, Abeona intends to address the requirements quickly.
Image Credit: Adobe Stock Images/M.Dörr & M.Frommherz
The FDA has issued a Complete Response Letter (CRL) in response to Abeona Therapeutics’ Biologics License Application (BLA) for pz-cel (prademagene zamikeracel), a treatment for recessive dystrophic epidermolysis bullosa (RDEB). According to the letter, the agency expressed a need for additional Chemistry Manufacturing and Controls (CMC) information for the drug to receive approval. Despite the rejection, there were no issues reported in safety or efficacy; however, there were issues reported with manufacturing and release testing methods validation.1
“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, Abeona, in a press release. “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.”
In late January, the FDA conducted a Bioresearch Monitoring and held a BLA mid-cycle review meeting, with the inspector not reporting any significant observations.2
“The Abeona team is committed to working with the FDA in its review of the pz-cel BLA, with the goal of bringing this therapy to patients as soon as possible,” said Seshadri, in a press release.
Back in November, the FDA accepted Abeona’s BLA for pz-cel and granted it Priority Review. The initial BLA was supported by promising results from the Phase III VITAL study, with additional support from a Phase I/II study. According to results from both studies, application of pz-cel displayed wound healing and pain reduction on chronic and large wounds.3
“The FDA’s acceptance of our BLA for priority review underscores the high unmet need in RDEB and the potential for pz-cel to provide meaningful benefit to these patients,” said Seshadri, in a press release. “We thank the FDA for their commitment and look forward to working with them through the BLA review, with the goal of bringing this therapy to patients as soon as possible.”
According to Medline Plus, dystrophic epidermolysis bullosa is prevalent in approximately 3.3 million people. It can cause fragile skin and make it easy for blisters to form. In some cases, disfiguring scars and disabling musculoskeletal deformities occur.4,5
“Dystrophic epidermolysis bullosa is one of the major forms of a group of conditions called epidermolysis bullosa,” reports Medline Plus. “Epidermolysis bullosa cause the skin to be very fragile and to blister easily. Blisters and skin erosions form in response to minor injury or friction, such as rubbing or scratching. The signs and symptoms of dystrophic epidermolysis bullosa vary widely among affected individuals. In mild cases, blistering may primarily affect the hands, feet, knees, and elbows. Severe cases of this condition involve widespread blistering that can lead to vision loss, scarring, and other serious medical problems.”
References
1. Abeona Therapeutics Provides Regulatory Update on Pz-cel. Abeona. April 22, 2024. Accessed April 24, 2024. https://investors.abeonatherapeutics.com/press-releases/detail/276/abeona-therapeutics-provides-regulatory-update-on-pz-cel
2. Abeona Therapeutics Announces Progress Update on Pz-cel Biologics License Application (BLA). Abeona. February 1, 2024. Accessed April 24, 2024. https://investors.abeonatherapeutics.com/press-releases/detail/271/abeona-therapeutics-announces-progress-update-on-pz-cel
3. Abeona Therapeutics Announces FDA Accepts and Grants Priority Review for Pz-cel Biologics License Application (BLA). Abeona. November 27, 2023. Accessed April 24, 2024. https://investors.abeonatherapeutics.com/press-releases/detail/268/abeona-therapeutics-announces-fda-accepts-and-grants
4. Dystrophic epidermolysis bullosa. MedlinePlus. Accessed April 24, 2024. https://medlineplus.gov/genetics/condition/dystrophic-epidermolysis-bullosa/
5. Epidermolysis Bullosa. NORD. Accessed April 24, 2024. https://rarediseases.org/rare-diseases/epidermolysis-bullosa/
