NEW YORK – Two gene therapies for treating a rare skin condition, Abeona Therapeutics' Zevaskyn (prademagene zamikeracel) and Krystal Biotech's Vyjuvek (beremagene geperpavec), recently garnered approval in the US and EU, respectively.
The US Food and Drug Administration has approved Abeona's Zevaskyn, a gene therapy to treat wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB), the company said Tuesday.
RDEB is a rare, debilitating skin disease caused by mutations in the COL7A1 gene that hinder production of functioning type VII collagen, which is needed for strengthening the layers of the skin.
Zevaskyn, formerly known as pz-cel, uses a retroviral vector to incorporate a functional collagen-producing COL7A1 gene into autologous skin cells that have been extracted from the patient, which are then surgically applied back to wound areas. Zevaskyn represents the first FDA-approved product to treat RDEB with a single application, Abeona said.
The FDA approved Zevaskyn based on data from Abeona's pivotal Phase III VIITAL trial, in which 81 percent of large, chronic wounds healed 50 percent or more after six months in patients who received the gene therapy, compared to 16 percent of matched control wounds treated with standard care. After gene therapy, patients also had less pain.
The most common adverse events, which were observed in fewer than 5 percent of patients, included procedural pain and itch.
"We are confident in Zevaskyn's ability to deliver long-term results," Madhav Vasanthavada, Abeona's chief commercial officer, said in a statement. "We are committed to working closely with both commercial and government payors on outcome-based agreements that stand behind the promise of Zevaskyn for patients, and expedite access."
Zevaskyn will be made available through qualified treatment centers in the third quarter, the company said. Cleveland-headquartered Abeona also will launch Abeona Assist to offer support for eligible patients and families, such as helping them understand their insurance benefits, financial assistance options, and travel and logistics assistance.
In connection with the FDA approval, Abeona received a rare pediatric disease priority review voucher, which it said it plans to monetize.
Separately, the European Commission last week granted marketing authorization to Pittsburgh-based Krystal Biotech for Vyjuvek, a gene therapy designed to treat wounds in patients with dystrophic epidermolysis bullosa (DEB) due to mutations in the COL7A1 gene.
Vyjuvek, which is already approved in the US, is delivered topically once a week as a gel and uses a genetically modified herpes-simplex virus type 1 vector to deliver two functional copies of COL7A1 to patients' wounds. Vyjuvek is the first corrective medicine approved in Europe to treat DEB, according to Krystal, and can be applied either at home or in a healthcare setting.
The EC's marketing authorization was based on data from the Phase I/II GEM-1 and Phase III GEM-3 clinical trials of Vyjuvek, as well as evidence from the company's open-label extension study and real-world data in the US. In the Phase III trial, after six months, Vyjuvek completely healed 67 percent of wounds, whereas 22 percent of the wounds healed for those administered placebo.
"This approval marks a critical milestone in our commitment to improving the lives of DEB patients around the world, and we look forward to providing patients in Europe with the first corrective medicine for this debilitating disease," Suma Krishnan, Krystal's president of R&D, said in a statement.
Vyjuvek will be made available in all EU member countries, as well as in Iceland, Norway, and Liechtenstein, though the timing of the gene therapy's availability in specific countries will depend on multiple factors, including completion of reimbursement procedures. Krystal expects to launch Vyjuvek in the first European country, Germany, mid-year.
The gene therapy is currently under regulatory review in Japan, and Krystal expects a decision in the second half of 2025.