Beremagene geperpavec

Zevaskyn is applied as genetically engineered skin grafts to treat chronic wounds in RDEB patients. Credit: Eder Paisan via Getty Images. Abeona Therapeutics has secured US Food and Drug Administration (FDA) approval for Zevaskyn (prademagene zamikeracel), a gene-corrected cell therapy designed to treat recessive dystrophic epidermolysis bullosa (RDEB). The approval follows years of clinical development and regulatory setbacks, and positions Abeona as the second company to bring a therapy for this condition to the US market. Also known as pz-cel, Zevaskyn is indicated for both paediatric and adult patients with RDEB. The therapy is administered as credit card-sized sheets of skin made from the patient’s own keratinocytes, which are harvested, genetically modified to express the functional COL7A1 gene, and surgically applied to chronic wounds. The COL7A1 gene encodes a type of collagen essential for anchoring skin layers – something patients with RDEB lack due to genetic mutations. The FDA had previously rejected an application from Abeona last year, citing outstanding manufacturing and quality control (QC) concerns. Following a complete response (CR) letter and further chemistry, manufacturing and controls (CMC) submissions, the agency granted approval based on results from a pivotal Phase III study (NCT04227106). Despite a partial clinical hold in 2019 , the study met both co-primary endpoints of wound healing and pain reduction, with no serious treatment-related adverse events (AEs) reported. The company also provided confirmatory evidence from a Phase I/IIa trial (NCT01263379), which supported the durability of healing after a single application. Zevaskyn is priced at $3.1m, placing it among the most expensive therapies in the US but in line with other rare disease gene and cell therapies. Abeona expects to make the product available commercially in Q3 2025. The company raised $35m in late 2022 after announcing the Phase III data, followed by a $25m financing round in July 2024 to support manufacturing and launch readiness. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence Krystal Biotech’s Vyjuvek (beremagene geperpavec), a topical gene therapy gel for DEB, was approved in 2023 and generated $290.5m in revenue in 2024. Unlike Zevaskyn, which is applied once through surgery, Vyjuvek is dosed weekly and costs around $631,000 per patient per year. Vyjuvek also delivers a functional COL7A1 gene, though via a re-dosable, non-integrating viral vector. In the 29 April announcement, Abeona’s CEO Vish Seshadri, said: “We have heard from the RDEB community that there is a persistent unmet need to reliably address RDEB wounds, especially those that are chronic and prone to infection. Through a single surgical application, Zevaskyn can now offer people with RDEB the opportunity for wound healing and pain reduction in even the most severe wounds, as evidenced by the results from our pivotal Phase III study.” In connection with the approval, Abeona received a rare paediatric disease priority review voucher (PRV), which the company said it plans to monetise. The PRV programme, which grants expedited FDA review for a future drug application, is set to expire soon unless renewed by Congress. Rare disease advocates have warned that the programme’s lapse , combined with existing FDA resource constraints, could deter future investment in paediatric rare disease research and development.

Abeona Therapeutics received FDA approval for its cell therapy to treat “butterfly skin,” a rare genetic disease that causes fragile skin and wounds. And its price tag makes it one of the most expensive therapies in the world. The cell therapy, known generically as pz-cel, will be marketed as Zevaskyn. It comes as credit card-sized skin sheets which are applied via surgery. It’s indicated for children and adults with recessive dystrophic epidermolysis bullosa, or RDEB, a skin disease that’s caused by a mutation in a gene for a type of collagen that keeps the skin layers together. The agency rejected the therapy last year over manufacturing questions. Abeona said Zevaskyn will cost $3.1 million, which puts its list price in range with other rare disease gene therapies. It is expected to be available in the third quarter of the year. The first therapy in the US to treat dystrophic epidermolysis bullosa was approved in 2023. Known as Vyjuvek, that treatment is sold by Krystal Biotech. It generated $290 million in revenue last year. That therapy costs about $631,000 per patient per year. Unlike Zevaskyn, Vyjuvek is administered once weekly. Both treatments have their roots in research conducted at Stanford, though Abeona says its therapy can potentially address more skin area than Vyjuvek can. Vyjuvek is a gene therapy that’s applied as a gel, while Zevaskyn is made from the patient’s own cells, which are harvested and then engineered to express the functional version of the collagen that people with DEB lack. In an interview, Abeona CEO Vishwas Seshadri said the company can manufacture 12 credit card-sized sheets at a time for each patient. Unlike typical cell and gene therapies, which are one-time treatments, the DEB treatments can be readministered. Abeona’s therapy was only administered once in clinical trial results reported so far. In a Phase 3 study, it reported that the cell therapy helped heal or shrink wounds in 11 children and adults. However, Seshadri said that in an ongoing study, patients were retreated with the skin sheets, though those data have not yet been shared publicly. In April 2024, the FDA issued a complete response letter to Abeona’s cell therapy. Seshadri said Abeona needed a faster test for sterility, which it then developed. The test is used to measure whether there are unwanted microorganisms, because its cell therapy has a short shelf life of just 84 hours. The company’s manufacturing site is in Cleveland. Along with the approval, Abeona gets a priority review voucher, which it could sell for over $100 million. As it stands right now, the priority review voucher program for rare pediatric disease is sunsetting, but rare disease advocates have pleaded with Congress to reauthorize the program, which they say provides an incentive, and in some cases a lifeline, for biotech companies to develop treatments for rare diseases.