Vyjuvek (beremagene geperpavec-svdt) / Krystal Biotech - LARVOL DELTA

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

Expansion of an HSV-1-based gene therapy platform to treat Hailey-Hailey and Darier diseases (SID 2025) - "The herpes simplex virus type 1 (HSV-1)-based gene therapy beremagene geperpavec (B-VEC) has been successful in treating the rare genetic skin blistering disease dystrophic epidermolysis bullosa (DEB)...Twenty-four hours after administration, skin punches were taken for DNA and RNA quantification, IF, and hematoxylin and eosin staining. In vitro and in vivo analyses indicated that both KB111 and KB112 are capable of transducing keratinocytes and expressing their encoded ATPases with minimal toxicity both in culture and in murine skin, demonstrating that Krystal's HSV-1-based platform is well-suited for the treatment of both HHD and DD."

Gene therapy • Dermatology • Gene Therapies • Genetic Disorders • Herpes Simplex

A randomized controlled trial of a novel skin substitute on epidermolysis bullosa wounds (SID 2025) - "The topical drugs, beremagene-geperpavec and birch-triterpenes, have been approved for RDEB wound healing but are not curative...No AEs were related to Spincare® and its discontinuation. Results from this pilot trial show that Spincare matrix treatment on RDEB wounds is a safe and tolerable cell therapy delivery method for a future Phase 1 trial of gene-edited induced pluripotent stem cells."

Clinical • Anemia • Dermatology • Hematological Disorders • Infectious Disease • Oncology • Pain • Squamous Cell Carcinoma

A Targeted Literature Review of Value-Based Agreements (VBAs) for Cell and Gene Therapies in the United States (ISPOR 2025) - "The therapies with VBAs in place included: Beqvez, Casgevy, Hemgenix, Kymriah, Luxturna, Lyfgenia, Roctavian, Vyjuvek, Zolgensma, and Zynteglo... In this review, multiple VBAs for CGTs were identified across multiple disease areas. Most payers did not publicly disclose which outcomes measures the VBAs were assessing. Of those that did, outcomes assessed could be sourced from routine patient visits and/or adjudicated claims, placing no additional burden on providers to collect data for the sole purpose of the VBA."

Gene therapy • Review • Gene Therapies

Deconstructing FDA's Expedited Program Determinations: Analyzing how the Agency may Set the Bar for RMAT Designation (ASGCT 2025) - "Parexel explored the relationship between the early-phase data from press releases around the time of RMAT designation and pivotal data from FDA review memos for marketing applications for two cases of RMAT-designated gene therapies, VYJUVEK (beremagene geperpavec) and BEQVEZ (fidanacogene elaparvovec-dzkt), that were approved in 2023 and 2024. Conducting analyses, such as the one listed above, that contextualize the RMAT-designation supporting data within the totality of publicly disclosed information for that development program, can help inform developers in making decisions about the amount of evidence and timing for submission of RMAT designation requests. Disease Focus of Abstract:Other Other: Rare Disease"

Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Krystal Biotech Announces First Quarter 2025 Financial and Operating Results (GlobeNewswire) - "$88.2 million in 1Q VYJUVEK revenue and $429.4 million since launch in Q3 2023...VYJUVEK (beremagene geperpavec-svdt, or B-VEC) for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)...The Company continues to expect a decision by Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) on its Japan New Drug Application (JNDA) in 2H 2025...The Company continues to enroll Cohort 3 of CORAL-1, the Company’s multi-center, dose escalation study evaluating KB407 in patients with CF...The Company received full sanctioning of the study protocol by the Cystic Fibrosis Foundation Therapeutic Development Network in January and remains on track for an interim molecular data readout for Cohort 3 patients in mid-2025...The Company continues to enroll in Cohort 2 and is working to enroll in Cohort 3 of SERPENTINE-1....The Company expects to report molecular results for the additional Cohort 2 and 3 patients later this year."

Enrollment status • Japan approval • P1 data • Sales • Alpha-1 Antitrypsin Deficiency • Cystic Fibrosis • Dermatology • Genetic Disorders

Krystal Biotech Announces European Commission Approval of VYJUVEK for the Treatment of Dystrophic Epidermolysis Bullosa (GlobeNewswire) - "Krystal Biotech, Inc...announced today that on April 23, 2025, the European Commission (EC) granted marketing authorization to VYJUVEK (beremagene geperpavec-svdt) for the treatment of wounds in patients with dystrophic epidermolysis bullosa (DEB) who have mutations in the collagen type VII alpha 1 chain (COL7A1) gene, starting from birth....This EC decision authorizes the marketing of VYJUVEK in all European Union member states, as well as Iceland, Norway and Liechtenstein....The Company is planning for its first European launch in Germany in mid-2025....The EC approval follows the positive recommendation issued by the European Medicines Agency’s Committee for Medicinal Products for Human Use in February and was based on a comprehensive clinical dataset including results from the Company’s Phase 1/2 GEM-1 and Phase 3 GEM-3 studies..."

EMA approval • Launch Europe • Dermatology • Genetic Disorders

Impact of COL7A1 Gene Therapy on SCC Recurrence in RDEB Skin (clinicaltrials.gov) - P2 | N=16 | Recruiting | Sponsor: Stanford University | Not yet recruiting ➔ Recruiting

Enrollment open • Gene Therapies • Oncology • Squamous Cell Carcinoma

Long-Term Safety and Tolerability of Beremagene Geperpavec-svdt (B-VEC) in an Open-Label Extension Study of Patients with Dystrophic Epidermolysis Bullosa. (PubMed, Am J Clin Dermatol) - P3 | "Patients undergoing extended B-VEC treatment maintained high satisfaction and continued to respond to treatment with no new safety signals detected in the open-label extension study, supporting the continuous use of B-VEC."

Journal • Gene Therapies • Herpes Simplex • COL7A1

VYJUVEK for Dystrophic Epidermolysis Bullosa: Ethical Dilemmas in Access and Pricing. (PubMed, J Am Acad Dermatol) - No abstract available

Journal • Pricing • Dermatology

Beremagene Geperpavec is the First FDA-Approved Topical Gene Therapy for Dystrophic Epidermolysis Bullosa (AAD 2025) - "Serious adverse events, not believed to be directly related to B-VEC, included hospitalizations for diarrhea, severe anemia, cellulitis, and infection related to a hemodialysis catheter. While there are no comparator trials with other therapies, B-VEC has been shown to be a safe and effective treatment for DEB in patients 6 months and older."

Gene therapy • Anemia • Dermatology • Gene Therapies • Hematological Disorders • Infectious Disease • Oncology • Pruritus • Squamous Cell Carcinoma • COL7A1

Krystal Biotech Receives Positive CHMP Opinion for VYJUVEK for the Treatment of Dystrophic Epidermolysis Bullosa (GlobeNewswire) - "Krystal Biotech, Inc...today welcomed the European Medicines Agency's (EMA’s) announcement that its Committee for Medicinal Products for Human Use (CHMP) has issued a positive recommendation for the European Commission (EC) to approve VYJUVEK (beremagene geperpavec-svdt, or B-VEC) for the treatment of wounds in patients with dystrophic epidermolysis bullosa (DEB) who have mutations in the collagen type VII alpha 1 chain (COL7A1) gene, starting from birth...The final EC decision is anticipated in the second quarter of 2025...The positive opinion issued by the CHMP is based on a comprehensive clinical dataset including results from the Company’s Phase 1/2 GEM-1 and Phase 3 GEM-3 studies..."

CHMP • EMA approval • Dermatology • Genetic Disorders

Impact of COL7A1 Gene Therapy on SCC Recurrence in RDEB Skin (clinicaltrials.gov) - P2 | N=16 | Not yet recruiting | Sponsor: Stanford University

Gene therapy • New P2 trial • Gene Therapies • Oncology • Squamous Cell Carcinoma

Estimating Costs in Beremagene Geperpavec for Dystrophic Epidermolysis Bullosa-Reply. (PubMed, JAMA Dermatol) - No abstract available

Journal