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FDA Grants Abeona Therapeutics Priority Review for Wound Healing Cell Therapy Application

NEW YORK – The US Food and Drug Administration has accepted for priority review Abeona Therapeutics' biologics license application seeking approval for an autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa (RDEB).

Cleveland-based Abeona is developing prademagene zamikeracel, or pz-cel for short, as a treatment for the rare connective tissue disorder caused by mutations in the COL7A1 gene, which hinder production of functioning type VII collagen needed for strengthening the layers of the skin. Patients with RDEB have severe, painful skin wounds that can cause systemic consequences and diminish quality of life.

Pz-cel uses a retroviral vector to incorporate a functional collagen-producing COL7A1 gene into patients' own skin cells. Abeona is hoping to show through clinical trials that this one-time treatment will facilitate normal gene expression over the long term and heal wounds and reduce pain in RDEB patients. 

In its BLA, Abeona submitted data on pz-cel's efficacy and safety from the pivotal Phase III VIITAL study and long-term follow-up and quality-of-life data from another Phase I/IIa study. According to the firm, in both studies, applying the cell therapy on large and chronic wounds in RDEB patients promoted wound healing and reduced pain.

In granting priority review to Abeona's BLA, the agency is slated to decide whether to grant market approval to pz-cel by May 25, 2024. The FDA does not plan to hold an advisory committee meeting for this application, according to Abeona. Pz-cel also has rare pediatric disease, orphan drug, breakthrough therapy, and regenerative medicine advance therapy designations from the FDA.

Cell and gene therapies for wound healing disorders are an active area of drug development. Earlier this year, the FDA approved Krystal Biotech's topical gene therapy Vyjuvek (beremagene geperpavec-svdt) for treating wounds in pediatric and adult patients with COL7A1-mutated dystrophic epidermolysis bullosa. Healiva and C4U also recently said they will jointly develop CRISPR-Cas3-based drugs for epidermolysis bullosa.