NEW YORK – The US Food and Drug Administration has issued a complete response letter (CRL) in response to Abeona Therapeutics' biologics license application (BLA) for an investigational autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa (RDEB), the firm said Monday.
The FDA accepted Cleveland-based Abeona's application for the cell therapy, known as prademagene zamikeracel, or pz-cel for short, and granted it priority review in November. As such, the FDA said it expected to decide whether to grant market approval for the cell therapy by May 25.
However, in its CRL, the FDA said it needed additional chemistry manufacturing and controls (CMC) information related to validation requirements for certain manufacturing and release testing methods, including some captured in observations during the FDA's pre-license inspection, the company said.
"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," Abeona CEO Vish Seshadri said in a statement. "We are already hard at work generating the additional CMC information."
Abeona anticipates submitting the requested CMC information to the FDA in Q3.
The CRL follows a late-cycle review meeting between the FDA and Abeona in March, in which the FDA had said additional information was needed to satisfy CMC requirements. Abeona previously said it would provide some CMC data before a potential BLA approval, as well as full validation reports in mid-2024. However, in its most recent letter, the FDA indicated that the proposed timing would not allow it sufficient time to complete its BLA review by May 25.
The letter did not identify deficiencies related to clinical efficacy or safety data submitted in the BLA, and did not request additional clinical studies, Abeona said.
Pz-cel uses a retroviral vector to incorporate a functional collagen-producing COL7A1 gene into patients' own skin cells in an effort to treat RDEB, a rare connective tissue disorder that's caused by mutations in the COL7A1 gene. These mutations hinder production of functioning type VII collagen needed for strengthening the layers of the skin.
The one-time cell therapy treatment is designed to facilitate normal gene expression and heal the severe, painful skin wounds associated with the disease.