1st RDEB patient in US to soon receive gene therapy Zevaskyn
Developer cites strong demand, with over a dozen people ID'd for treatment
The first recessive dystrophic epidermolysis bullosa (RDEB) patient will be dosed with the cell-based gene therapy Zevaskyn (prademagene zamikeracel) in the next few months, with several others lined up to follow, according to developer Abeona Therapeutics.
That patient has already been selected, and their skin cells have been collected to generate the therapy, which is made on-demand for each individual.
“Zevaskyn’s launch is demonstrating positive early momentum,” Vish Seshadri, CEO of Abeona Therapeutics, said in a company press release. “The first Zevaskyn patient treatment is on track for the third quarter of 2025, with multiple additional patients identified and advancing through the process to initiate treatment.”
U.S. regulators approved Zevaskyn in April for the treatment of wounds in adults and children with RDEB. The therapy is now available at two qualified treatment centers — the Ann & Robert H. Lurie Children’s Hospital of Chicago and Lucile Packard Children’s Hospital Stanford in California.
Abeona indicated there’s been strong demand, with more than a dozen eligible patients identified for treatment at these two centers and several advancing through the administrative process.
More than three dozen other patients have been identified as candidates for treatment at referring sites that are not qualified treatment centers. The company also expects to activate additional treatment sites this year.
To address the growing interest in Zevaskyn, Abeona is ramping up its supply capacity, and remains on track to have enough Zevaskyn to treat up to 10 patients per month in mid-2026.
Abeona working with insurers to help make sure patients have coverage
The company has also been working with public and private insurers to make sure that patients will have adequate coverage for the one-time gene therapy, which comes with a list price of more than $3 million.
Multiple large national and regional payers have agreed to cover the therapy. United Healthcare, the largest commercial insurer in the U.S., published a favorable policy for Zevaskyn coverage that’s consistent with its prescribing label and without additional restrictions.
Abeona has also entered into the National Drug Rebate Agreement with the U.S. Centers for Medicare and Medicaid Services (CMS), which will help obtain expedited coverage of Zevaskyn across Medicaid programs in all states and Puerto Rico. The company indicated some states have already implemented favorable coverage criteria.
So far, 100% of prior authorization requests for Zevaskyn submitted to insurers have been approved, according to Abeona.
A patient support program called Abeona Assist is available to help patients and families who are considering Zevaskyn. It offers information about the treatment pathway, financial assistance, and travel and logistical support.
“The enthusiasm from the RDEB community and clinicians, alongside our substantial progress with payer coverage, affirms ZEVASKYN’s crucial role in transforming patient care,” Seshadri said.
Zevaskyn treatment involves collecting patient’s skin cells, engineering them
RDEB is caused by mutations in the COL7A1 gene, which leads to a deficiency in the collagen type VII (COL7) protein that helps skin maintain structural integrity. This gives rise to fragile skin that easily tears and blisters, along with chronic wounds that don’t heal normally.
Zevaskyn treatment involves collecting a patient’s skin cells and engineering them in the lab to carry a healthy version of COL7A1. The engineered cells are grown onto a thin sheet that’s surgically grafted onto a patient’s wounds for long-term, local production of COL7 to promote healing.
The therapy’s approval was supported by the Phase 3 VIITAL trial (NCT04227106), final data from which were published in The Lancet earlier this summer.
VIITAL enrolled 11 people with RDEB, each of whom had some wounds treated with Zevaskyn and others that were left untreated.
The results showed that after six months, 81% of Zevaskyn-treated wounds were at least 50% healed, which was significantly more than the 16% of untreated wounds that were at least 50% healed. These results met one of the study’s main goals.
Zevaskyn also led to significant reductions in wound pain, meeting its other main goal, and was well tolerated.
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