FDA Approves Abeona Therapeutics’ First and Only Gene Therapy for RDEB Wounds

CLEVELAND, April 29, 2025 (GLOBE NEWSWIRE) — Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced the U.S. Food and Drug Administration (FDA) has approved ZEVASKYN™ (prademagene zamikeracel) gene-modified cellular sheets, also known as pz-cel, as the first and only autologous cell-based gene therapy for treating wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB), a severe genetic skin disease. Currently, there is no cure for RDEB, and ZEVASKYN is the only FDA-approved product to treat RDEB wounds with a single application.

“Today’s approval of ZEVASKYN is a pivotal moment for the RDEB community, addressing a significant unmet need for reliable wound treatment,” said Vish Seshadri, Ph.D., M.B.A., Chief Executive Officer of Abeona. “A single surgical application of ZEVASKYN offers the potential for wound healing and pain reduction in even the most severe RDEB wounds, as demonstrated in our Phase 3 study.”

First-of-its-kind gene therapy with robust clinical evidence

The FDA approval of ZEVASKYN is based on the positive results from the pivotal Phase 3 VIITAL™ study (NCT04227106), a multi-center, randomized, intrapatient-controlled trial. The study met its co-primary endpoints, showing statistically significant healing of ≥50% in large chronic RDEB wounds and pain reduction from baseline (Wong-Baker FACES scale) at six months post-treatment.

In 43 large and chronic wounds treated with a single ZEVASKYN application, 81% achieved ≥50% healing at six months (P<0.0001), compared to 16% of matched control wounds treated with standard of care. The most common adverse events, occurring in <5% of patients, were procedural pain and itch.

“ZEVASKYN was well-tolerated and efficacious in clinical studies, providing clinically meaningful improvements in wound healing, pain reduction, and associated symptoms in large chronic RDEB wounds after a single application,” said Jean Tang, M.D., Ph.D., lead principal investigator of the VIITAL™ study. “Long-term improvements in wound healing and pain reduction have been observed for years after a single ZEVASKYN application in the Phase 1/2a study. Today marks the availability of a promising new therapeutic option made possible by the courageous participation of patients and families in these studies.”

The Phase 1/2a study (NCT01263379) showed long-term improvement at treated sites over a median follow-up of 6.9 years (range 4-8 years) in 38 chronic wounds across 7 patients after a single ZEVASKYN application.

“After many years of work, this FDA approval of ZEVASKYN is a significant achievement for EB patients,” said M. Peter Marinkovich, M.D., co-principal investigator of the VIITAL™ study.

“The strong data from our clinical trials gives us confidence in ZEVASKYN’s ability to deliver long-term results after a single treatment,” said Madhav Vasanthavada, Ph.D., M.B.A., Chief Commercial Officer of Abeona. “We are committed to working with payers on outcome-based agreements to ensure patient access to ZEVASKYN.”

ZEVASKYN was well-tolerated across both clinical studies, with no treatment-related serious adverse events observed.

Anna L. Bruckner, MD, Co-Director of the EB Clinic at Children’s Hospital of Colorado, stated, “The FDA approval of ZEVASKYN is a monumental step forward, offering a much-needed, long-lasting treatment option and hope for improved quality of life for individuals with RDEB and their families.”

Amy Paller, M.D., pediatric dermatologist and clinical researcher, commented, “Grafting gene-corrected skin offers the potential for long-term healing, pain reduction, and reduced infection risk for chronic RDEB wounds. This therapy nicely complements recently approved topical products.”

Dr. Seshadri added, “We are deeply grateful to the patients, families, and caregivers who supported ZEVASKYN’s development. We thank debra of America for their unwavering support and advocacy for the EB community. We also acknowledge the clinical investigators, study site personnel, and the entire Abeona team for their dedication to this milestone. We look forward to providing the RDEB community access to ZEVASKYN.”

Addressing the underlying cause of RDEB

RDEB results from mutations in both copies of the COL7A1 gene, leading to a deficiency in Type VII collagen, causing extremely fragile skin with extensive blistering and severe, often chronic wounds. These wounds cause debilitating pain and systemic complications, impacting lifespan and quality of life.

ZEVASKYN consists of a patient’s own skin cells (keratinocytes) genetically modified to produce functional Type VII collagen. These ZEVASKYN sheets are surgically applied to wounds, with up to 12 credit card-sized sheets able to cover large areas or multiple wounds in a single application.

Brett Kopelan, M.A., Executive Director of debra of America, said, “The FDA’s approval of ZEVASKYN is very exciting. Given its potential to address even the largest and most challenging chronic wounds, we believe ZEVASKYN can significantly improve the quality of life for patients. It has the potential to transform the day-to-day standard of care for these debilitating wounds, reducing pain and stress for both patients and caregivers. We are honored to partner with Abeona and look forward to ensuring broad patient access to ZEVASKYN.”

Michael Hund, M.B.A., Chief Executive Officer of EB Research Partnership (EBRP), stated, “EBRP is dedicated to funding research for EB treatments and cures. We commend Abeona’s leadership and commitment to delivering innovative solutions for the EB community. ZEVASKYN’s approval is a landmark moment, and Abeona’s leadership in gene therapy holds great promise for many rare diseases. EBRP looks forward to continued collaboration with Abeona.”

ZEVASKYN availability in the third quarter of 2025

ZEVASKYN is expected to be available in the third quarter of 2025 through ZEVASKYN Qualified Treatment Centers (QTCs), located across the U.S. Abeona’s patient support program, Abeona Assist™, offers personalized support, including insurance and financial assistance information, and travel and logistical assistance for eligible patients. For more information, patients, caregivers, and providers can call Abeona Assist at 1-855-ABEONA-1 (1-855-223-6621) or email [email address removed].

“The development of ZEVASKYN was made possible by the dedication of scientists over the past decade,” said Marissa Perman, MD, Section Chief of Dermatology at Children’s Hospital of Philadelphia. “Having a new, uniquely differentiated gene therapy for RDEB patients is a significant milestone, offering the potential for fuller, less painful, and less itchy lives with fewer wounds. I look forward to seeing its impact in our practice.”

Joyce Teng, MD, PhD, professor in dermatology, added, “The approval of ZEVASKYN is a groundbreaking advancement for recessive dystrophic EB, a condition that has long needed innovative solutions. ZEVASKYN offers additional hope for affected patients and families and represents a scientific triumph in improving quality of life. We look forward to seeing its impact.”

In connection with the FDA approval, Abeona received a Rare Pediatric Disease Priority Review Voucher (PRV), which the Company plans to monetize.