Zevaskyn (prademagene zamikeracel) / Stanford University, Abeona Therap - LARVOL DELTA

Update on Approved Therapies for Dystrophic and Junctional Epidermolysis Bullosa. (PubMed, Pediatr Dermatol) - "Since 2022, three therapies - birch triterpenes gel (Filsuvez), beremagene geperpavec-svdt (B-VEC, Vyjuvek), and prademagene zamikeracel (pz-cel, Zevaskyn) - have received regulatory approval, representing the first specific interventions for epidermolysis bullosa. Herein, we provide an overview on their mechanisms of action, efficacy, safety, and clinical implications."

Journal • Review • Dermatology • Gene Therapies

Phase 4 Master Protocol for Patients Prescribed Prademagene Zamikeracel for the Treatment of Wounds (clinicaltrials.gov) - P4 | N=100 | Not yet recruiting | Sponsor: Abeona Therapeutics, Inc

New P4 trial

A Long-Term Safety Follow-up Registry for Patients Who Received Treatment With Prademagene Zamikeracel for Recessive Dystrophic Epidermolysis Bullosa (RDEB) in the Post-Marketing Setting (clinicaltrials.gov) - P=N/A | N=250 | Recruiting | Sponsor: Abeona Therapeutics, Inc

New trial

Sustained wound healing and long-term safety of prademagene zamikeracel in recessive dystrophic epidermolysis bullosa (RDEB): Five-year results from the VIITAL™ phase 3 trial (SID 2026) - "The mean (±SD) pain reduction assessed by the investigator using the validated Wong-Baker FACES Scale was greater for treated wounds versus control wounds at all months. Long-term follow-up through five years demonstrated an overall safety profile for pz-cel that was consistent with observations from the primary study phase, with no new safety signals identified"

Clinical • P3 data • COL7A1

Long-term safety and clinical outcomes of pz-cel gene therapy in a single patient with recessive dystrophic epidermolysis bullosa: A 12-year case report (SID 2026) - "Prademagene zamikeracel (pz-cel) is an autologous cell sheet-based gene therapy that was US-FDA approved in April 2025 for pediatric and adult patients with recessive dystrophic epidermolysis bullosa (RDEB)A 23-year-old male with RDEB enrolled in a Phase 1/2a clinical trial and underwent surgical application of pz-cel in October 2013, followed by a long-term follow-up study with 12 years of safety data available...No replication-competent retrovirus or SCC was observed at treated sites. This case demonstrates durable wound healing and a favorable long-term safety profile following pz-cel treatment, with sustained clinical benefit and no treatment-related serious adverse events over 12 years of follow-up, approaching the FDA- recommended 15-year monitoring period for cell and gene therapies"

Case report • Clinical • Clinical data • Gene therapy • Corneal Abrasion • Gene Therapies • Hematological Disorders • Squamous Cell Carcinoma

SPENDING ON GENE THERAPIES FOR RECESSIVE DYSTROPHIC EPIDERMOLYSIS BULLOSA (ISPOR 2026) - "OBJECTIVES: The Food and Drug Administration (FDA) has recently approved two gene therapies for recessive dystrophic epidermolysis bullosa (RDEB): beremagene geperpavec (B-VEC; Vyjuvek), a once-weekly drug with a yearly cost of $657,000 (approved in 2023), and prademagene zamikeracel (PZ-CEL; Zevaskyn), a one-time therapy costing $3.1 million (approved in 2025). This analysis quantifies the trade-offs that payers face when balancing up-front costs with those accruing over longer durations for high-priced gene therapies in RDEB and may consider alternative payment mechanisms or new negotiation models."

Gene therapy • Gene Therapies

An evaluation of prademagene zamikeracel for the treatment of recessive dystrophic epidermolysis bullosa. (PubMed, Expert Opin Biol Ther) - "This review outlines the key scientific foundations, translational advances, and clinical trial outcomes that supported regulatory approval of prademagene zamikeracel for use in adult and pediatric patients with RDEB. The development of prademagene zamikeracel represents a significant advance in regenerative therapy for RDEB, demonstrating that durable restoration of C7 expression and long-term wound improvement can be achieved through skin grafting."

Journal • Review • Non-melanoma Skin Cancer • Oncology • Pain • Pediatrics • Squamous Cell Carcinoma • Squamous Cell Skin Cancer • COL7A1

FDA approval of prademagene zamikeracel (Zevaskyn): advancing gene therapy for recessive dystrophic epidermolysis bullosa (RDEB) patients. (PubMed, Ann Med Surg (Lond)) - No abstract available

FDA event • Journal • Gene Therapies

Prademagene Zamikeracel: First Approval. (PubMed, Mol Diagn Ther) - "Prademagene zamikeracel contains the patient's own genetically modified cells with functional copies of the COL7A1 gene, as patients lacking functional copies of this gene may develop chronic open wounds caused by the separation of dermal layers. This article summarizes the milestones in the development of prademagene zamikeracel leading to this first approval for treatment of wounds in adult and paediatric patients with recessive dystrophic epidermolysis bullosa."

Journal • Gene Therapies • Pediatrics • COL7A1

Prademagene zamikeracel (Zevaskyn) for recessive dystrophic epidermolysis bullosa. (PubMed, Med Lett Drugs Ther) - No abstract available

Journal

Abeona Therapeutics Closes Sale of Rare Pediatric Disease Priority Review Voucher for $155 Million (The Brussels Times) - "Abeona Therapeutics Inc...announced the closing of the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $155 million on June 27, 2025. Including net proceeds from the sale of the PRV, the Company reported that unaudited cash, cash equivalents, restricted cash and short-term investments as of June 30, 2025 were approximately $225 million....'The PRV proceeds, combined with our existing cash, provides Abeona with robust financial flexibility, ensuring over two years of operating capital for sustained growth without the need for further capital infusion and prior to accounting for ZEVASKYN sales. We anticipate the first ZEVASKYN patient treatment in Q3 2025, with profitability projected for early 2026'."

Commercial • Genetic Disorders

A Phase 3b Study for the Treatment of Dystrophic Epidermolysis Bullosa (DEB) in New and Previously EB-101 Treated Patients (clinicaltrials.gov) - P3 | N=12 | Active, not recruiting | Sponsor: Abeona Therapeutics, Inc | Recruiting ➔ Active, not recruiting | Trial completion date: Jun 2025 ➔ Sep 2025 | Trial primary completion date: Mar 2025 ➔ Sep 2025

Enrollment closed • Trial completion date • Trial primary completion date

Prademagene zamikeracel for recessive dystrophic epidermolysis bullosa wounds (VIITAL): a two-centre, randomised, open-label, intrapatient-controlled phase 3 trial. (PubMed, Lancet) - P3 | "Prademagene zamikeracel improved wound healing and pain versus control and was well tolerated, supporting its potential to reduce wound burden in patients with large, chronic RDEB wounds."

Journal • P3 data • Dermatology • Pain • COL7A1

Prademagene Zamikeracel. (PubMed, Am J Health Syst Pharm) - No abstract available

Journal

Absence of insertional oncogenesis and RCR in clinical and pre-clinical experience with prademagene zamikeracel, a retrovirally delivered cell-based gene therapy, in recessive dystrophic epidermolysis bullosa patients (SID 2025) - "In clinical trial subjects, no SCC was observed at 144 pz-cel-treated sites yet occurred exclusively in untreated areas. In SCC cases, RCR and proviral genome testing results were negative. In an ex-vivo non-clinical study, six RDEB samples were retrovirally transduced yielding 3, 203, 395 reads, 193, 317 inferred cells, and 184, 931 unique integration sites."

Gene therapy • Late-breaking abstract • Preclinical • Gene Therapies • COL7A1

Presence of large progenitor cell populations in pz-cel autologous gene-corrected epidermal sheets (SID 2025) - "Pz-cel (prademagene zamikeracel) was developed to address large, chronic wounds in RDEB patients... These results confirm the presence of a significant population of progenitor cells within the graft. The large proportion of these cells in the drug product may increase the likelihood of wound site repopulation by long-lasting, genetically corrected cells, consistent with the detection of Col7 at grafted sites at 24 months."

Clinical • Late-breaking abstract • Genetic Disorders • COL7A1 • ITGB1 • TFRC

Abeona Therapeutics Enters into Agreement to Sell Priority Review Voucher for $155 Million (GlobeNewswire) - "Abeona Therapeutics Inc...today announced it has entered into a definitive asset purchase agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $155 million upon the closing of the transaction. Abeona was awarded the PRV following the U.S. Food and Drug Administration (FDA) approval of ZEVASKYN (prademagene zamikeracel) on April 28, 2025....The transaction is subject to customary closing conditions, including expiration of the applicable waiting period under the Hart-Scott Rodino (HSR) Antitrust Improvements Act."

Commercial • Genetic Disorders

U.S. FDA Approves ZEVASKYN (prademagene zamikeracel), the First and Only Cell-Based Gene Therapy for Patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB) (GlobeNewswire) - "Abeona Therapeutics...today announced the U.S. Food and Drug Administration (FDA) has approved ZEVASKYN (pronounced as ‘ZEE-vah-skin’) (prademagene zamikeracel) gene-modified cellular sheets, also known as pz-cel, as the first and only autologous cell-based gene therapy for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB), a serious and debilitating genetic skin disease....The FDA approval of ZEVASKYN is based on the pivotal Phase 3 VIITAL study (NCT04227106)..."

FDA approval • Genetic Disorders

Abeona Therapeutics Reports Full Year 2024 Financial Results, Provides Pz-cel Regulatory Update and Commercial Launch Plans (GlobeNewswire) - "On March 14, 2025, Abeona received draft United States Prescribing Information (USPI) from the FDA to initiate discussion on the label for pz-cel. Discussions are also ongoing with the FDA on post-approval marketing requirements and commitments for pz-cel. If approved, the Company anticipates the first patient treatment with pz-cel in the third quarter of 2025...Obtained two additional patents from the United States Patent and Trademark Office for pz-cel, extending patent protection on the use of pz-cel for the treatment of RDEB to June 2037 and patent protection on the packaging and transport system for pz-cel to July 2040."

FDA event • Launch US • Patent • Genetic Disorders • Rare Diseases

Abeona Therapeutics Announces FDA Acceptance of BLA Resubmission of Pz-cel for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (GlobeNewswire) - "Abeona Therapeutics Inc...announced that the U.S. Food and Drug Administration (FDA) has accepted for review Abeona’s resubmission of its Biologics License Application (BLA) for prademagene zamikeracel (pz-cel), its investigational autologous cell-based gene therapy, as a potential new treatment for recessive dystrophic epidermolysis bullosa (RDEB). The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of April 29, 2025."

FDA filing • PDUFA • Genetic Disorders • Rare Diseases

Abeona Therapeutics Announces Favorable Medicare Reimbursement Decisions for Pz-cel (GlobeNewswire) - "Abeona Therapeutics Inc...announced that the Centers for Medicare and Medicaid Services (CMS) has granted a product-specific procedure code ICD-10-PCS (International Classification of Diseases, 10th Revision, Procedure Coding System) for prademagene zamikeracel (pz-cel), Abeona’s investigational autologous cell-based gene therapy currently in development for recessive dystrophic epidermolysis bullosa (RDEB). If pz-cel receives U.S. marketing approval, this code will allow for efficient and accurate documentation, billing, and analysis of inpatient hospital procedures using pz-cel. The code will go into effect on October 1, 2024."

Reimbursement • Genetic Disorders • Rare Diseases

EB-101 Treatment for New and Previously Treated Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB) (clinicaltrials.gov) - P3 | N=12 | Recruiting | Sponsor: Abeona Therapeutics, Inc | Phase classification: P3b ➔ P3 | Trial completion date: Dec 2024 ➔ Jun 2025 | Trial primary completion date: Aug 2024 ➔ Mar 2025

Phase classification • Trial completion date • Trial primary completion date

Eicosapentaenoic Acid Level Predicts Long-Term Survival and Cardiovascular or Limb Event in Peripheral Arterial Disease. (PubMed, Ann Vasc Dis) - "In Cox stepwise multivariate analysis, lower EPA (hazard ratio [HR]: 0.996, 95% confidence interval [CI]: 0.993-1.000, p = 0.034), ankle brachial pressure index (ABI), body mass index, serum albumin, eGFR, age, CRP, D-dimer, critical limb ischemia, diabetes, cerebrovascular disease (CVD), and statin were related to ACD (p <0.05); lower EPA (HR: 0.997, 95% CI: 0.994-1.000, p = 0.038), ABI, serum albumin, eGFR, age, diabetes, coronary heart disease, CVD, and statin were related to MACEs (p <0.05); and lower EPA (HR: 0.988, 95% CI: 0.982-0.993, p <0.001), ABI, and low-density lipoprotein cholesterol were related to LEAEs (p <0.05). Low plasma EPA level was a significant risk factor for ACD, MACEs, and LEAEs in patients with PAD."

Journal • Cardiovascular • CNS Disorders • Coronary Artery Disease • Diabetes • Dyslipidemia • Heart Failure • Metabolic Disorders • Peripheral Arterial Disease • Vascular Neurology • CRP

Exploring the Financial Burden Associated with the Management of Dystrophic Epidermolysis Bullosa (DEB): A Systematic Literature Review (SLR) with Database Review of Future Disease-Modifying Therapies (DMTs) (ISPOR 2024) - "Nine clinical trials research 7 potential DMTs: advanced/gene therapies (EB-101, B-VEC, ALLO-ASC-SHEET, FCX-007), protein-replacement therapies (PTR-01, PTR-01-002), and a proteinase inhibitor (Polyphenon E). Previously, as there were no DMT options for patients with DEB treatment was symptomatic only. Future innovative DMTs should aim to prevent skin wounds, thereby decreasing the need for hospitalization and outpatient visits."

Review • Dermatology • Gene Therapies

EB-101 Treatment for New and Previously Treated Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB) (clinicaltrials.gov) - P3b | N=12 | Recruiting | Sponsor: Abeona Therapeutics, Inc | Active, not recruiting ➔ Recruiting

Enrollment open