FDA Sets PFUDA Date, Accepts BLA Submission for Pz-cel in Epidermolysis Bullosa

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Abeona Therapeutics announced today that the US Food and Drug Administration (FDA) has accepted its resubmitted Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) for the treatment of recessive dystrophic epidermolysis bullosa.¹

The FDA has established a Prescription Drug User Fee Act (PDUFA) target action date of April 29, 2025.

Abeona's initial BLA for the COL7A1 gene-corrected epidermal sheets was submitted to the FDA in September 2023.² The FDA accepted the BLA and granted it priority review the following month, establishing an initial PDUFA date of May 24, 2024.³

However, earlier this year, the FDA issued a Complete Response Letter in response to the BLA⁴ just one month shy of the anticipated target action date.

The FDA identified the need for additional Chemistry, Manufacturing, and Controls information, including validation for specific manufacturing and release testing methods noted during the pre-license inspection. However, the CRL did not find deficiencies in the clinical efficacy or safety data of pz-cel.

Last month, Abeona resubmitted its BLA for pz-cel.⁵

The BLA's acceptance is based on positive data from the pivotal phase 3 VIITAL study (NCT04227106) and supporting phase 1/2a data (NCT01263379), which demonstrated sustained wound healing and pain reduction with pz-cel.

"The FDA acceptance of our BLA resubmission moves us one step closer to providing pz-cel as a differentiated treatment option to address the persistent unmet needs of people with RDEB in the US," said Vish Seshadri, chief executive officer of Abeona, in a news release.¹ "We look forward to continuing to work with the FDA to finalize the review of the pz-cel application."

References

1. Abeona Therapeutics announces FDA acceptance of BLA resubmission of Pz-cel for the treatment of recessive dystrophic epidermolysis bullosa. News release. Globe Newswire. November 12, 2024. Accessed November 12, 2024. https://www.globenewswire.com/news-release/2024/11/12/2978973/0/en/Abeona-Therapeutics-Announces-FDA-Acceptance-of-BLA-Resubmission-of-Pz-cel-for-the-Treatment-of-Recessive-Dystrophic-Epidermolysis-Bullosa.html
2. Abeona Therapeutics submits Biologics License Application to US FDA seeking Priority Review and approval of EB-101 for the treatment of patients with recessive dystrophic epidermolysis bullosa. Abeona Therapeutics Inc. September 26, 2023. Accessed November 12, 2024. https://investors.abeonatherapeutics.com/press-releases/detail/265/abeona-therapeutics-submits-biologics-license-application.
3. Abeona Therapeutics announces FDA accepts and grants Priority Review for pz-cel biologics license application (BLA). Abeona Therapeutics. News release. November 27, 2023. Accessed November 12, 2024. https://investors.abeonatherapeutics.com/press-releases/detail/268/abeona-therapeutics-announces-fda-accepts-and-grants
4. Abeona Therapeutics provides regulatory update on pz-cel. News release. Abeona Therapeutics. April 22, 2024. Accessed November 12, 2024. https://investors.abeonatherapeutics.com/press-releases/detail/276/abeona-therapeutics-provides-regulatory-update-on-pz-cel
5. Abeona Therapeutics completes pz-cel Biologics License Application resubmission to US Food and Drug Administration. News release. Abeona Therapeutics. October 29, 2024. Accessed November 12, 2024. https://investors.abeonatherapeutics.com/press-releases/detail/291/abeona-therapeutics-completes-pz-cel-biologics-license