Krystal Biotech Receives Positive CHMP Opinion for VYJUVEK® for the Treatment of Dystrophic Epidermolysis Bullosa
Krystal Biotech (NASDAQ: KRYS) has received a positive recommendation from the European Medicines Agency's CHMP for VYJUVEK®, a treatment for dystrophic epidermolysis bullosa (DEB). The therapy targets patients with COL7A1 gene mutations from birth.
The CHMP's recommendation supports VYJUVEK administration in clinical settings or at home, with trained patients or caregivers allowed to apply the treatment. The European Commission's final approval decision is expected in Q2 2025, covering EU member states, Iceland, Norway, and Liechtenstein.
The recommendation is based on Phase 1/2 GEM-1 and Phase 3 GEM-3 studies, published in Nature Medicine and the New England Journal of Medicine. The company plans to launch first in Germany mid-2025, followed by France later that year.
Krystal Biotech (NASDAQ: KRYS) ha ricevuto una raccomandazione positiva dal CHMP dell'Agenzia Europea dei Medicinali per VYJUVEK®, un trattamento per l'epidermolisi bollosa distrofica (DEB). La terapia è destinata ai pazienti con mutazioni del gene COL7A1 fin dalla nascita.
La raccomandazione del CHMP supporta l'amministrazione di VYJUVEK in contesti clinici o a casa, consentendo ai pazienti o ai caregiver formati di applicare il trattamento. La decisione finale di approvazione da parte della Commissione Europea è attesa nel secondo trimestre del 2025, coprendo gli stati membri dell'UE, Islanda, Norvegia e Liechtenstein.
La raccomandazione si basa sugli studi di Fase 1/2 GEM-1 e di Fase 3 GEM-3, pubblicati su Nature Medicine e New England Journal of Medicine. L'azienda prevede di lanciare per prima cosa in Germania a metà 2025, seguita dalla Francia più tardi nello stesso anno.
Krystal Biotech (NASDAQ: KRYS) ha recibido una recomendación positiva del CHMP de la Agencia Europea de Medicamentos para VYJUVEK®, un tratamiento para la epidermólisis bullosa distrófica (DEB). La terapia está dirigida a pacientes con mutaciones del gen COL7A1 desde el nacimiento.
La recomendación del CHMP respalda la administración de VYJUVEK en entornos clínicos o en casa, permitiendo que pacientes o cuidadores capacitados apliquen el tratamiento. Se espera que la decisión final de aprobación de la Comisión Europea se tome en el segundo trimestre de 2025, abarcando a los estados miembros de la UE, Islandia, Noruega y Liechtenstein.
La recomendación se basa en los estudios de Fase 1/2 GEM-1 y de Fase 3 GEM-3, publicados en Nature Medicine y en el New England Journal of Medicine. La empresa planea lanzar primero en Alemania a mediados de 2025, seguido de Francia más tarde ese mismo año.
크리스탈 바이오텍 (NASDAQ: KRYS)는 유럽 의약품청의 CHMP로부터 피부 박리증(EPB) 치료제 VYJUVEK®에 대한 긍정적인 권고를 받았습니다. 이 요법은 COL7A1 유전자 변이가 있는 환자를 대상으로 하며, 출생 시부터 적용됩니다.
CHMP의 권고는 임상 환경이나 가정에서 VYJUVEK의 사용을 지원하며, 훈련된 환자나 보호자가 치료를 적용할 수 있도록 허용합니다. 유럽연합 집행위원회의 최종 승인 결정은 2025년 2분기에 예상되며, EU 회원국, 아이슬란드, 노르웨이, 리히텐슈타인을 포함합니다.
이 권고는 Nature Medicine 및 New England Journal of Medicine에 발표된 1/2상 GEM-1 및 3상 GEM-3 연구에 기반합니다. 회사는 2025년 중반에 독일에서 최초로 출시할 계획이며, 그 해 후반에 프랑스에서 출시할 예정입니다.
Krystal Biotech (NASDAQ: KRYS) a reçu une recommandation positive du CHMP de l'Agence européenne des médicaments pour VYJUVEK®, un traitement de l'épidermolyse bulleuse dystrophique (DEB). La thérapie cible les patients présentant des mutations du gène COL7A1 depuis la naissance.
La recommandation du CHMP soutient l'administration de VYJUVEK dans des contextes cliniques ou à domicile, permettant aux patients ou aux aidants formés d'appliquer le traitement. La décision finale d'approbation de la Commission européenne est attendue au deuxième trimestre 2025, couvrant les États membres de l'UE, l'Islande, la Norvège et le Liechtenstein.
La recommandation est basée sur les études de Phase 1/2 GEM-1 et de Phase 3 GEM-3, publiées dans Nature Medicine et le New England Journal of Medicine. L'entreprise prévoit de lancer d'abord en Allemagne à la mi-2025, suivie par la France plus tard dans l'année.
Krystal Biotech (NASDAQ: KRYS) hat eine positive Empfehlung vom CHMP der Europäischen Arzneimittel-Agentur für VYJUVEK®, eine Behandlung für dystrophische Epidermolysis bullosa (DEB), erhalten. Die Therapie richtet sich an Patienten mit COL7A1-Genmutationen seit der Geburt.
Die Empfehlung des CHMP unterstützt die Verabreichung von VYJUVEK in klinischen Einrichtungen oder zu Hause, wobei geschulte Patienten oder Pflegepersonen die Behandlung anwenden dürfen. Die endgültige Genehmigungsentscheidung der Europäischen Kommission wird im zweiten Quartal 2025 erwartet und umfasst die EU-Mitgliedstaaten, Island, Norwegen und Liechtenstein.
Die Empfehlung basiert auf den Studien der Phase 1/2 GEM-1 und Phase 3 GEM-3, die in Nature Medicine und dem New England Journal of Medicine veröffentlicht wurden. Das Unternehmen plant, zunächst Mitte 2025 in Deutschland auf den Markt zu kommen, gefolgt von Frankreich später in diesem Jahr.
- First-ever corrective therapy for DEB patients in Europe
- Broad label approval from birth
- Flexible administration options (clinic/home)
- Geographic expansion beyond US market
- Real-world efficacy data from US launch supports approval
- Launch timeline extends to Q2 2025
- Phased rollout across Europe may delay full market penetration
Insights
The positive CHMP opinion for VYJUVEK represents a significant regulatory milestone for Krystal Biotech, essentially clearing the path for European Commission approval in Q2 2025. This recommendation would enable commercialization across the EU plus Iceland, Norway, and Liechtenstein—substantially expanding the addressable market for this first-in-class gene therapy.
The favorable broad label recommendation is particularly noteworthy, including treatment from birth and the flexibility of home administration by trained patients or caregivers. These provisions significantly enhance the therapy's commercial potential by removing access barriers and expanding the eligible patient population to include even infants.
Strategically, Krystal has positioned itself for a rapid European rollout, with Germany targeted for mid-2025 and France later in the year. The company's commercial infrastructure development appears well-timed to capitalize on approval. The demonstration of durable wound closure through gene delivery represents the first corrective therapy for DEB, a condition with treatment options, giving Krystal a strong market position backed by compelling clinical evidence published in prestigious journals.
VYJUVEK's mechanism directly addresses the genetic defect underlying DEB rather than just treating symptoms, representing a paradigm shift in treatment approach with potential applications to other genetic skin conditions in Krystal's pipeline.
This regulatory advancement significantly strengthens Krystal's commercial outlook, as European market access would substantially expand VYJUVEK's revenue potential beyond its current US operations where it launched in 2023. The positive CHMP recommendation typically translates to EC approval with approximately
The market opportunity is particularly valuable given VYJUVEK's first-mover advantage for DEB, an orphan disease with high unmet need. While rare disease therapies target small populations, they command premium pricing that can drive substantial revenues. The home administration provision is financially significant as it dramatically improves the therapy's accessibility and reduces healthcare system costs, factors that can positively influence reimbursement negotiations with European payers.
Krystal appears well-prepared for commercialization with an established European leadership team and staged country launch sequence prioritizing Germany—Europe's largest pharmaceutical market with typically favorable rare disease reimbursement. This methodical approach maximizes early revenue capture while building infrastructure for broader expansion.
With market capitalization of approximately
EC approval decision anticipated in second quarter of 2025
PITTSBURGH, Feb. 28, 2025 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company, 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 CHMP’s positive opinion includes support for administration of VYJUVEK in either a health care setting (e.g., a clinic) or at home. If deemed appropriate by a healthcare professional, trained patients or caregivers may also apply VYJUVEK.
The final EC decision is anticipated in the second quarter of 2025. The decision will be applicable to all European Union member states, as well as Iceland, Norway and Liechtenstein.
“We are excited to be able to provide DEB patients with the first treatment that corrects the genetic defect and makes a true difference in their lives,” said Cristina Has, M.D., Professor and Head of the Genodermatoses Clinic in the Department of Dermatology at the University of Freiburg in Germany. “By addressing the very first stage in the complex pathophysiology of DEB, VYJUVEK is a landmark. It is amazing how simple and non-invasive its use is, even in infants.”
“We are very pleased that our patients, from birth, will have a simple, topical treatment that promotes durable wound closure, something that until now has been beyond the reach of any therapy,” added Christine Bodemer, M.D., PhD, Professor and Head of the Department of Dermatology at the Necker Enfants Malades Hospital in Paris. “This is a remarkable advance for DEB patients and a new approach to gene therapy for genodermatoses, revolutionary and remarkably innovative.”
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, published in Nature Medicine and the New England Journal of Medicine, respectively, which collectively provided clear clinical evidence of successful COL7A1 gene delivery and durable wound closure following topical administration. The long-term safety and efficacy of B-VEC is further supported by results from the Company’s open label extension study completed in the United States as well as real-world experience with VYJUVEK since launching the United States in 2023.
“The CHMP’s recommendation for approval of VYJUVEK is an exciting step towards our goal of delivering the first ever corrective therapy to DEB patients across Europe,” said Suma Krishnan, President of Research and Development at Krystal Biotech. “The CHMP’s support for a broad label, including treatment of patients from birth and the option of patient or caregiver administration at home, are also fantastic outcomes for the DEB patients we aim to serve, broadening access and reducing barriers to starting on and staying on therapy.”
If approved by the EC, the Company expects to market B-VEC under the registered European trademark, VYJUVEK.
“Our team in Europe has been working tirelessly in preparation for Krystal’s first commercial launch outside of the United States and with the CHMP’s positive opinion we remain on track to launch in Germany around the middle of this year,” said Laurent Goux, Senior Vice President and General Manager of Europe at Krystal Biotech. “This will be the first of many launches in Europe, including a launch in France planned for later in 2025, as we advance diligently to ensure as many patients as possible benefit from access to VYJUVEK.”
About VYJUVEK
VYJUVEK is a non-invasive, topical, redosable gene therapy designed to deliver two copies of the COL7A1 gene when applied directly to DEB wounds. VYJUVEK was designed to treat DEB at the molecular level by providing the patient’s skin cells the template to make normal COL7 protein, thereby addressing the fundamental disease-causing mechanism. VYJUVEK was approved in the United States by the U.S. Food and Drug Agency (FDA) in May 2023 for the treatment of wounds in patients six months of age or older with DEB.
The use of VYJUVEK in the European Union remains investigational.
U.S. INDICATION
VYJUVEK is a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy indicated for the treatment of wounds in patients six months of age and older with dystrophic epidermolysis bullosa with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene.
U.S. IMPORTANT SAFETY INFORMATION
Adverse Reactions
The most common adverse drug reactions (incidence >
To report SUSPECTED ADVERSE REACTIONS, contact Krystal Biotech, Inc. at 1-844-557-9782 or FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch.
Contraindications
None.
Warnings and Precautions
VYJUVEK gel must be applied by a healthcare provider.
After treatment, patients and caregivers should be careful not to touch treated wounds and dressings for 24 hours.
Wash hands and wear protective gloves when changing wound dressings. Disinfect bandages from the first dressing change with a virucidal agent, and dispose of the disinfected bandages in a separate sealed plastic bag in household waste. Dispose of the subsequent used dressings in a sealed plastic bag in household waste.
Patients should avoid touching or scratching wound sites or wound dressings.
In the event of an accidental exposure flush with clean water for at least 15 minutes.
For more information, see full U.S. Prescribing Information.
About Krystal Biotech, Inc.
Krystal Biotech, Inc. (NASDAQ: KRYS) is a commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs. VYJUVEK® is the Company’s first commercial product, the first-ever redosable gene therapy, and the first medicine approved by the FDA for the treatment of dystrophic epidermolysis bullosa. The Company is rapidly advancing a robust preclinical and clinical pipeline of investigational genetic medicines in respiratory, oncology, dermatology, ophthalmology, and aesthetics. Krystal Biotech is headquartered in Pittsburgh, Pennsylvania. For more information, please visit http://www.krystalbio.com, and follow @KrystalBiotech on LinkedIn and X (formerly Twitter).
Forward-Looking Statements
Any statements in this press release about future expectations, plans and prospects for Krystal Biotech, Inc., including statements about the expected timing of the European Commission approval of VYJUVEK, the expectation that the Company will market B-VEC in the European Union under the registered European trademark, VYJUVEK, the timing of expected commercial launches in Germany and France, and other statements containing the words “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “target,” “potential,” “likely,” “will,” “would,” “could,” “should,” “continue,” and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including uncertainties associated with applications for marketing approvals and other important factors as are set forth under the caption “Risk Factors” in the Company’s annual and quarterly reports on file with the U.S. Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company’s views as of the date of this press release. The Company anticipates that subsequent events and developments will cause its views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company’s views as of any date subsequent to the date of this press release.
CONTACT
Investors and Media:
Stéphane Paquette, PhD
Krystal Biotech
spaquette@krystalbio.com
FAQ
When will VYJUVEK be available in Europe after CHMP's positive opinion for KRYS?
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