Vyjuvek (beremagene geperpavec-svdt) / Krystal Biotech - 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

Engineering HSV-1 for Gene Therapy, Oncolytic Immunotherapy, and Neuroscience. (PubMed, Virol Sin) - "Clinical successes such as T-VEC, G47Δ, and B-VEC have validated the clinical potential of HSV-1 engineering, yet broader translation remains limited by antiviral immunity, inefficient delivery, epigenetic silencing, genome instability, and manufacturing challenges. In this review, we illustrate how HSV-1 has evolved from a naturally neurotropic virus into a versatile biomedical tool, whose therapeutic and research potential emerges from the precise matching of viral properties with disease-specific requirements, delivery contexts, and functional objectives."

Journal • Review • Gene Therapies • Herpes Simplex

Topical gene therapy for severe recessive dystrophic epidermolysis bullosa: clinical outcomes and multidisciplinary management in three patients treated with beremagene geperpavec (B-VEC) in Italy - a case series. (PubMed, Drugs Context) - "Adjunctive care included wound hygiene optimization, nutritional and iron support, and pain control. In this small descriptive case series, B-VEC was well tolerated and associated with clinically meaningful improvement in wound healing though outcomes were more variable and, in some cases, less pronounced than those reported in pivotal trials, nevertheless supporting its integration into a comprehensive care model that addresses infection risk, anaemia, nutrition, and wound management."

Clinical data • Journal • Fibrosis • Gene Therapies • Hematological Disorders • Immunology • Infectious Disease • Pain • COL7A1

What is the formula for commercial success of a gene therapy? Reflections on approved GTx (ASGCT 2026) - "Indication selection: Commercial success across Casgevy®, Vyjuvek®, Elevidys® and Zolgensma® reinforce five criteria that appear pivotal for gene therapy success...Beqvez® (hemophilia B) appears to have treated no patients between FDA approval (2024) and market withdrawal (2025), while Hemgenix® treated 12 patients in its first year (<0.1% penetration). Uptake has also remained minimal for Zevaskyn® and Zynteglo® (beta-thalassemia).By contrast, Elevidys® (DMD) treated approximately 900 patients in the US within two years of FDA approval, although it did not receive EU authorization... The number of clinical study initiations increased continuously over the observation period, rising by 430% from 27 ongoing company sponsored programs in 2016 to 143 in 2025. After steady growth until 2021, development activity accelerated markedly from 2022 onward, with the pipeline almost doubling between 2022 and 2023. After a peak in 2024,..."

Gene therapy • Beta-Thalassemia • CNS Disorders • Duchenne Muscular Dystrophy • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Hepatology • Liver Failure • Metabolic Disorders • Movement Disorders • Muscular Atrophy • Muscular Dystrophy • Ophthalmology • Rare Diseases

Treatment of aplasia cutis congenita in an infant with severe recessive dystrophic epidermolysis bullosa using beremagene geperpavec (SID 2026) - "This case highlights the safety, tolerability, and potential benefit of early BVEC therapy in infants with RDEB, suggesting that early intervention may promote effective wound healing, interrupt the chronic wound cycle, and improve outcomes in severe phenotypes. Further studies are warranted to evaluate the durability and immunologic implications of early gene therapy exposure."

Gene Therapies • Herpes Simplex • Inflammation • COL7A1

MHRA approves beremagene geperpavec (Vyjuvek) for the treatment of dystrophic epidermolysis bullosa (GOV.UK) - "Beremagene geperpavec is a gel, applied directly to wounds."

MHRA approval • Dermatology • Genetic Disorders

Topical B-VEC for Epidermolysis Bullosa Wounds: Early Epithelialization in a Pediatric and Adolescent Case Series (EADV-Sp 2026) - "Larger cohorts and longer follow-up are warranted to assess durability of response and long-term safety. EADV Symposium 2026 – Athens"

Clinical • Gene Therapies • Infectious Disease • Pediatrics • COL7A1

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

Real-World Use of the Topical HSV-1-Based Gene Therapy B-VEC by Dominant Dystrophic Epidermolysis Bullosa Patients (AAD 2026) - "Of patients with a severity for their DDEB reported, 47% were mild, 41% were moderate, and 11% were severe. As of the data cutoff in July 2025, more than 75% of patients remained on therapy, supportive of the benefit of B-VEC in the DDEB population."

Clinical • Gene therapy • Real-world • Real-world evidence • Gene Therapies • Herpes Simplex • COL7A1

Healing Dystrophic Epidermolysis Bullosa Wounds with Topical Gene Therapy: A Single-Arm Meta-Analysis of Beremagene Geperpavec (AAD 2026) - "B-VEC offers rapid, meaningful wound healing in DEB with minimal treatment-related adverse events. Despite limitations including few studies, heterogeneous designs, and a limited Japanese cohort, findings consistently show its efficacy and favorable safety, supporting B-VEC as a well-tolerated, potentially transformative therapy for DEB."

Gene therapy • Retrospective data • Gene Therapies • Genetic Disorders • Herpes Simplex • COL7A1

Understanding the efficacy and tolerability of topical therapies for epidermolysis bullosa. (PubMed, Expert Rev Clin Pharmacol) - "This review examines the clinical efficacy and tolerability of topical therapies for EB, with a particular focus on approved treatments such as beremagene geperpavec (B-VEC) and Oleogel-S10, as well as emerging agents like allantoin, diacerein, sirolimus, gentamicin, gentian violet and calcipotriol...The continued use of standardized outcome measures will affect the future progress of studies, as well as using real-world data. Whilst a definitive cure is unlikely for EB, evolving topical strategies have strong potential in providing control of the disease's symptoms and ultimately can improve long-term outcomes."

Journal • Review • Dermatology • Pain

Developing CRISPR-Based Therapies for Epidermolysis Bullosa: A Comprehensive Review of Current Strategies. (PubMed, Drugs) - "The recent US Food and Drug Administration (FDA)-approved in vivo gene replacement therapy beremagene geperpavec (Vyjuvek®) provides a promising solution, but it requires ongoing application and is not applicable to all forms of EB...Further, new tools including prime editing have recently emerged and hold significant potential for EB gene therapy. Ongoing developments in gene editing technologies focused on improving safety and editing precision offer significant promise for the future clinical translation of potentially lifelong treatments for people with EB."

Journal • Review • Gene Therapies

Scarring Alopecia in Localized Dystrophic Epidermolysis Bullosa: A Case Report and a Scoping Review. (PubMed, Cureus) - "In this report, we present a case of a 58-year-old female with an eight-year history of pretibial DEB and a documented heterozygous COL7A1 mutation, who presented to the dermatology clinic with improving skin fragility after five months of topical beremagene geperpavec therapy, now noting gradual hair thinning on the vertex scalp over one year...Together, these findings were consistent with scarring alopecia with shared features of DEB and lichen planopilaris (LPP). We highlight the importance of recognizing scarring alopecia as a complication of DEB, with discernment for concomitant inflammatory etiologies that may co-occur, especially in a patient with a COL7A1 mutation."

Journal • Alopecia • Dermatology • Fibrosis • Immunology • Lichen Planus • COL7A1

A Patient-Level Simulation to Bridge Data Gaps in Cost-Effectiveness Modeling of B-VEC for Dystrophic Epidermolysis Bullosa (ISPOR-EU 2025) - "Patient-level simulation offers a viable solution for modelling treatment impact and resource use in the absence of patient-level trial data. This approach may be particularly valuable in rare diseases like DEB, where traditional data sources are limited."

Clinical • Cost effectiveness • HEOR • Gene Therapies • Rare Diseases

The Saudi National Policy and Protocol for Epidermolysis Bullosa. (PubMed, Risk Manag Healthc Policy) - "In addition, the guideline discusses the role of new treatments for EB, such as Beremagene geperpavec (B-VEC), and stresses the necessity of creating a National Saudi EB Registry to maximize patient care and guarantee fair access to treatment. The guideline also emphasizes the importance of genetic counseling for prenatal diagnosis, family planning, and parental screening, as well as the need to address the psychological difficulties that people living with EB and their families face. In summary, this guideline offers a structured framework for physicians and healthcare providers to guarantee the best possible care for people living with EB in Saudi Arabia, with the goal of improving clinical outcomes, quality of life, and long-term management of the disease."

Journal • Dermatology

Off-Label Use of Double-Dose Beremagene Geperpavec (B-VEC) in the Treatment of Recessive Dystrophic Epidermolysis Bullosa: A Promising Clinical Case. (PubMed, Pediatr Dermatol) - "While B-VEC has shown significant promise in treating DEB, few studies have examined off-label use of B-VEC at higher dose volumes. We describe the successful off-label use of a temporarily increased B-VEC dose in a patient with recessive DEB (RDEB) with extensive wounds following an episode of acute urticaria, highlighting the potential utility of higher dose B-VEC in managing patients with extensive wound burden."

Journal • Dermatology • Gene Therapies • Herpes Simplex • Immunology • Urticaria

B-VEC Gene Therapy for Dystrophic Epidermolysis Bullosa: Real-world data from the treatment of 9 patients (EADV 2025) - "B-VEC is a well-tolerated therapy that has shown promising results in reducing wound area in this case series. However, multiple factors influence treatment success. Chronic wounds—particularly those on scarred background—require longer treatment durations, whereas acute wounds tend to heal fast and remain closed."

Clinical • Gene therapy • Real-world • Real-world evidence • Gene Therapies • Herpes Simplex • Infectious Disease • Squamous Cell Carcinoma • COL7A1

Krystal Biotech Announces FDA Approval of Updated VYJUVEK Label (GlobeNewswire) - "This label update is based on real-world data collected since VYJUVEK launch in the United States, as well as results from the open label extension study conducted in the United States and published earlier this year, which collectively reinforce the long-term safety and efficacy of VYJUVEK across patients of all ages, including in cases of patient or caregiver application."

FDA approval • Genetic Disorders

Beremagene geperpavec (B-VEC) for infantile dystrophic epidermolysis bullosa: Benefit of early intervention. (PubMed, J Eur Acad Dermatol Venereol) - No abstract available

Journal • Gene Therapies

Efficacy and Safety of the Topical Gene Therapy Beremagene Geperpavec-svdt (B-VEC) in an Open-Label Study of Japanese Subjects With Dystrophic Epidermolysis Bullosa. (PubMed, J Dermatol) - "The results of the Japan OLE study are in agreement with the US Phase 3 and OLE studies, demonstrating the efficacy and safety of B-VEC in Japanese patients with DEB. Trial Registration: Japan Registry of Clinical Trials: jRCT2053230075."

Journal • Gene Therapies • Herpes Simplex • Pain • COL7A1

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

Journal

Treatment progress of inherited epidermolysis bullosa. (PubMed, Expert Opin Biol Ther) - "PubMed was searched up to February 2025 to identify relevant studies on inherited EB. Emerging advances of therapies in gene-based therapy and cell-based therapy can partially address the symptoms of EB and provide hope for the patients."

Journal • Review • Dermatology • Gene Therapies • Mood Disorders • Pain

Towards Extracellular Vesicles in the Treatment of Epidermolysis Bullosa. (PubMed, Bioengineering (Basel)) - "Current treatments focus on symptomatic relief through wound care and pain management, with recent FDA approvals of Vyjuvek and Filsuvez providing new but limited therapeutic options...However, challenges remain, including standardization of EV production, scalability, and ensuring consistent therapeutic potency. Despite these hurdles, EV-based therapies represent a transformative step toward addressing the complex pathology of EB, with the potential to improve wound healing, reduce fibrosis, and enhance patient quality of life."

Journal • Review • Dermatology • Fibrosis • Graft versus Host Disease • Immunology • Oncology • Pain

IOLITE: A Study Assessing B-VEC Compared to Matching Placebo for the Treatment and Prevention of Corneal Abrasions in Dystrophic Epidermolysis Bullosa (clinicaltrials.gov) - P3 | N=16 | Recruiting | Sponsor: Krystal Biotech, Inc.

New P3 trial • Corneal Abrasion • Ophthalmology

Clinical characteristics of recessive dystrophic epidermolysis bullosa patients with collagen VII antibodies (SID 2025) - "Certain therapies may induce host production of C7 such as gentamicin, exon skipping, and beremagene geperpavec, or patients may be exposed to exogenous C7 such as gene-corrected grafting, intravenous recombinant C7, and bone marrow transplantation. The pathogenicity of these antibodies and their impact on therapy response in RDEB patients remains unclear and warrants further evaluation. Importantly, understanding C7 antibodies may assist future therapeutic research in RDEB patients."

Clinical • Bone Marrow Transplantation • Gene Therapies