Epidermolysis Bullosa Dystrophica

The US Food and Drug Administration (FDA) has approved Abeona Therapeutics’ Zevaskyn (prademagene zamikeracel) for use in recessive dystrophic epidermolysis bullosa (RDEB). The decision makes Zevaskyn the first autologous cell-based gene therapy approved in the US to treat wounds in adult and paediatric patients with the rare skin disorder. RDEB is an incurable connective tissue disease that causes the skin to become very fragile. It is characterised by extensive blistering and severe wounds that often cover more than 30% of a patient’s body surface. Zevaskyn consists of a patient’s own skin cells that are genetically modified to produce functional type VII collagen, which RDEB patients are unable to produce due to a defect in both copies of the COL7A1 gene. The therapy is surgically applied to a patient’s wounded areas in the form of a sheet and is expected to be available in the US from the third quarter of this year. Abeona’s chief executive officer, Vish Seshadri, said the approval represents a “pivotal moment in the treatment of RDEB”. “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,” Seshadri said. The FDA’s approval was supported by results from the phase 3 VIITAL study, which demonstrated significant wound healing and pain reduction after a single treatment with Zevaskyn. In the trial, 81% of the 43 large and chronic wounds treated with a single application of Abeona’s therapy showed 50% or more healing at six months, compared to 16% in 43 matched control wounds treated with standard of care. A phase 1/2a study in 38 chronic wounds across seven patients also showed that a single application of Zevaskyn was associated with long-term improvement at treated sites over a median follow-up of 6.9 years. Zevaskyn was well-tolerated in both studies, with no treatment-related serious adverse events observed to date. Madhav Vasanthavada, Abeona’s chief commercial officer, said: “Based on the strength of our data across clinical trials, we are confident in Zevaskyn’s ability to deliver long-term results after a single treatment application. “We are committed to working closely with both commercial and government payers on outcome-based agreements that stand behind the promise of Zevaskyn for patients, and expedite access.”

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.

EB Research Partnership ("EBRP") invested in the work that led to ZEVASKYN at Stanford University under their pioneering Venture Philanthropy Model. This approval marks a historic moment for the EB community as the third FDA approved treatment and the first autologous, cell-based gene therapy for RDEB (Recessive Dystrophic Epidermolysis Bullosa). NEW YORK, April 29, 2025 /PRNewswire/ -- In a significant advancement for the Recessive Dystrophic Epidermolysis Bullosa (RDEB) community, the U.S. Food and Drug Administration (FDA) has approved a third treatment for this rare condition. The milestone approval of Abeona Therapeutics' ZEVASKYN not only represents a continued commitment to innovation in RDEB care, but reinforces the growing momentum toward improved quality of life and expanded treatment choices for individuals and families affected by RDEB. ZEVASKYN is the first autologous, cell-based gene therapy for RDEB, and the first RDEB treatment designed to provide collagen VII expression at wound sites via a stably integrated copy of the COL7A1 gene. Continue Reading “For over 20 years, I’ve watched my son face the relentless challenges of EB with strength no child should ever have to summon,” says Faye Dilgen, EBRP Board Member and mother of John Hudson Dilgen, who lives with RDEB. “This new treatment doesn’t just offer new relief, hope, and expanded options, it brings us closer to a future where children with EB won’t have to endure the same struggles.” EB is a group of devastating and life-threatening skin disorders that affect children from birth. Children with EB are often called “Butterfly Children” because their skin is as fragile as the wings of a butterfly. A BREAKTHROUGH IS NOW A BLUEPRINT EB Research Partnership (EBRP) is the world's largest nonprofit funding life-changing research to accelerate treatments and find a cure for EB, while pioneering a path for rare disease at large. EB is one of over 10,000 rare diseases, 95% of which have no approved treatments or cures. Through investments in cutting-edge approaches like gene therapies, stem cell therapies, antibody treatments, and more, EBRP is driving scientific breakthroughs that have the potential to impact the lives of 400 million people around the world living with a rare disease. "For over 20 years, I've watched my son face the relentless challenges of EB with strength no child should ever have to summon," says Faye Dilgen, EBRP Board Member and mother of John Hudson Dilgen, who lives with RDEB. "This new treatment doesn't just offer new relief, hope, and expanded options, it brings us closer to a future where children with EB won't have to endure the same struggles. We are incredibly grateful to the scientists, advocates, and especially to Abeona and EB Research Partnership for their tireless dedication in making this day possible for families like ours and future generations." The game-changing approval of ZEVASKYN underscores how progress in EB can serve as a model for advancements across the rare disease space. "Abeona's development and advancement of ZEVASKYN delivers a landmark moment for the global EB community, and their leadership in gene therapy holds so much promise to innovate the therapeutic landscape for not only EB, but many other rare diseases and conditions," says Michael Hund, CEO of EBRP. A MODEL TO SCALE SOLUTIONS With this FDA approval, EBRP gains continued momentum in its mission to discover treatments and a cure for EB. This milestone reinforces the validity of their scalable, sustainable Venture Philanthropy model, and, most importantly, provides treatment options and ignites hope for families around the world affected by EB. EBRP invested in the ground-breaking work that led to ZEVASKYN at Stanford University under their Venture Philanthropy Model. The funding followed the highly competitive application and screening process overseen by EBRP's Scientific Advisory Board (SAB), which is composed of leading scientists and physicians. EBRP was able to realize a significant return on its investment and reinvest that capital back into additional promising EB research projects. EBRP's Venture Philanthropy business model has been highlighted for its leadership by Harvard Business School, Stanford Social Innovation Review, Yale University, the Milken Institute, and MIT. "EB Research Partnership is proud to be an early investor via our Venture Philanthropy Model in the science leading to ZEVASKYN at Stanford University. The collaboration between EBRP and Stanford exemplifies the mission of EBRP to advance commercially sustainable research aimed at treating and ultimately curing epidermolysis bullosa. We are honored to partner with the entire Abeona team and commend their leadership, determination, and passion to deliver much needed innovative solutions for individuals and their families living with EB. They continue to share our values and commitment to accelerate treatments to the EB community as quickly as possible," says Michael Hund, CEO of EBRP. A COURAGEOUS COMMUNITY DRIVING CHANGE EB is a group of devastating and life-threatening skin disorders that affect children from birth. Children with EB are often called "Butterfly Children" because their skin is as fragile as the wings of a butterfly. With skin this fragile, those with EB face severe pain, open external and internal wounds, and grueling bandaging processes. For the 500,000 people around the world living with EB, everyday activities like eating, sleeping, walking and playing can become monumental tasks that often require modification. Over the last decade, EBRP has made undeniable progress in its mission to find a cure for this community, including: Raising over $70M Funding 160+ EB projects Contributing to a 25x growth in the EB clinical trial landscape Directly funding two FDA-approved EB treatments Today's FDA approval of the first autologous, cell-based gene therapy for RDEB is an exciting proof point of this progress. EBRP will continue to build off this momentum and is dedicated to delivering a cure. A PERIOD OF UNPRECEDENTED AWARENESS ZEVASKYN's approval arrives during an unprecedented wave of awareness for EB, fueled by recent high-impact initiatives led by EBRP and their Co-Founders Eddie and Jill Vedder. Launched in 2025, the Vedder Cup is an annual series between the San Diego Padres and Seattle Mariners that carries a meaningful purpose beyond the game. Through their partnership with EBRP, both teams are actively driving increased awareness and visibility for Epidermolysis Bullosa. This collaboration is not only shining a national spotlight on EB but also advancing efforts to accelerate the development of treatments and a cure. The upcoming premiere of "Matter of Time", a compelling documentary capturing the electrifying energy of Eddie Vedder's October 2023 solo concerts in Seattle, is bringing new levels of EB visibility on a global scale. Premiering in June 2025 at the Tribeca Festival, "Matter of Time" weaves together powerful music and the moving stories of individuals impacted by EB - including families, researchers, and thought leaders - and highlights how innovation and determination are driving progress toward a cure. One such researcher chronicled throughout the film is Dr. Jean Tang of Stanford University, who was lead principal investigator on the clinical of this third FDA approved treatment. The documentary is a testament to the power of music, community, and unwavering determination to drive change and inspire hope. Public awareness of EB has never been stronger—and this most recent FDA approval underscores the momentum building around research, advocacy, and support. About EB Research Partnership Founded in 2010 by a dedicated group of parents along with Jill and Eddie Vedder of Pearl Jam, EB Research Partnership (EBRP) is the largest global nonprofit dedicated to funding research to accelerate treatments and find a cure for Epidermolysis Bullosa (EB), a group of devastating and life-threatening skin disorders that affect children from birth. When making a grant to a research project, EBRP utilizes a Venture Philanthropy model. They retain the added upside of generating a recurring revenue stream if the therapy or product is commercially successful, then use the return on investment to fund additional EB research until a cure is found. While EB is a rare disease, there are 10,000 rare diseases that affect 1 in 10 people in the world, 95% of which have no treatments, that can benefit from the research they fund and this innovative model. To learn more about EBRP, visit For more information on ZEVASKYN, visit For more information on Matter of Time, visit SOURCE EB Research Partnership WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED