FDA Approves $3.1M Gene Therapy for Rare, Painful Skin Disorder

The U.S. FDA has approved Zevaskyn from Aveona Therapeutics to treat recessive dystrophic epidermolysis bullosa, a rare skin condition.

In recessive dystrophic epidermolysis bullosa, the skin is incredibly fragile, resulting in blisters all over the body and large, difficult-to-heal wounds. When a COL7A1 gene mutation prevents the synthesis of essential collagen that joins the skin’s upper and lower layers, recessive dystrophic epidermolysis bullosa may result.

According to Aveona, 10 to 14 patients are anticipated to receive Zevaskyn treatment this year. Zevaskyn will be available at a few treatment facilities beginning in the third quarter of this year for $3.1 million.

In Zevaskin, a gene therapy, the patient’s skin cells are extracted, a healthy COL7A1 gene is added, and the skin cells are then transplanted back into the patient. Early- to mid-phase and late-phase clinical trials have demonstrated its efficacy in reducing pain and healing wounds.

Currently available treatments for FS include medication treatment with Crystal Biotech’s “VyJuvek,” approved in 2023, and symptomatic measures like daily wound care and bandaging. Bisubec, which was sold for $290.5 million last year, is used to treat minor wounds.

Experts anticipate patients can treat FS more successfully if they use Zevaskyn and VyJuvek simultaneously.