Persistence has paid off for Abeona Therapeutics and its gene therapy for recessive dystrophic epidermolysis bullosa (RDEB).
About a year after the FDA rejected the company's prior approval filing, Abeona announced today that the agency has approved Zevaskyn, its autologous cell-based gene therapy for adults and pediatric patients with RDEB.
RDEB is a serious and debilitating genetic skin disease that causes severe wounds to form on patients' skin. The wounds can cover more than 30% of a person's body surface, and sometimes as much as 80%, according to Abeona, causing debilitating pain and various other complications.
In a phase 3 trial dubbed VIITAL, the drug, also known as prademagene zamikeracel, showed an ability to heal large chronic RDEB wounds and relieve pain after 6 months. Specifically, across several dozen "large and chronic wounds" treated with a single Zevaskyn application, 81% of the wounds showed 50% or better healing. For the trial's standard of care comparison, 16% of matched wounds showed that level of healing after 6 months.
Patients with RDEB have mutations in both copies of the COL7A1 gene that expresses Type VII collagen, suffering from fragile skin that often blisters and breaks. The drug incorporates a patient's skin cells that have been genetically modified to produce functional Type VII collagen. The treatment is then applied as a "sheet" over wounds.
Last year, the FDA turned away the drug based on chemistry, manufacturing and controls (CMC) issues that needed to be addressed.
Now, with the approval, Abeona is gearing up for a commercial launch in the third quarter of this year through qualified treatment centers that will be spread around the U.S. to ensure patient access.
The company plans to establish "outcome-based agreements" with commercial and government payers that "stand behind the promise of Zevaskyn for patients," Abeona chief commercial officer Madhav Vasanthavada, Ph.D., said in a statement.
Abeona has further established a manufacturing facility in Cleveland that's slated to support the rollout.
The biotech received a rare pediatric disease priority review voucher along with the FDA nod. The company said it plans to monetize the voucher.
Elsewhere in the industry, Krystal Biotech is progressing with the launch of its own topical gene therapy in dystrophic epidermolysis bullosa. The drug, Vyjuvek, won its FDA nod in May 2023 and has generated more than $341 million since its launch, according to a Krystal presentation (PDF) last month.