Krystal Biotech Announces Approval of VYJUVEK® by Japan’s Ministry of Health, Labour and Welfare for the Treatment of Dystrophic Epidermolysis Bullosa
Krystal Biotech (NASDAQ: KRYS) has received marketing authorization from Japan's Ministry of Health, Labour and Welfare for VYJUVEK®, marking a significant milestone as the first genetic medicine approved in Japan for treating dystrophic epidermolysis bullosa (DEB).
The approval allows for flexible administration options, including home dosing and administration by patients or family members. VYJUVEK works by delivering functional copies of the human COL7A1 gene to provide wound healing and sustained functional type VII collagen protein expression. The company expects to launch in Japan by the end of 2025, pending completion of reimbursement procedures.
Clinical data supporting the approval included results from a Japanese open label extension study, where all four patients achieved full wound closure at six months, consistent with previous U.S. Phase 3 study results. The treatment has already received approvals in the United States (May 2023) and Europe (April 2025).
Krystal Biotech (NASDAQ: KRYS) ha ottenuto l'autorizzazione alla commercializzazione dal Ministero della Salute, Lavoro e Welfare del Giappone per VYJUVEK®, segnando un traguardo importante come primo medicinale genetico approvato in Giappone per il trattamento della epidermolisi bollosa distrofica (DEB).
L'approvazione consente opzioni di somministrazione flessibili, inclusa la somministrazione domiciliare da parte dei pazienti o dei loro familiari. VYJUVEK agisce fornendo copie funzionali del gene umano COL7A1 per favorire la guarigione delle ferite e l'espressione sostenuta della proteina collagene di tipo VII funzionale. L'azienda prevede di lanciare il prodotto in Giappone entro la fine del 2025, in attesa del completamento delle procedure di rimborso.
I dati clinici a supporto dell'approvazione includono i risultati di uno studio di estensione a etichetta aperta condotto in Giappone, in cui tutti e quattro i pazienti hanno ottenuto la completa chiusura delle ferite a sei mesi, in linea con i risultati precedenti dello studio di Fase 3 negli Stati Uniti. Il trattamento ha già ricevuto l'approvazione negli Stati Uniti (maggio 2023) e in Europa (aprile 2025).
Krystal Biotech (NASDAQ: KRYS) ha recibido la autorización de comercialización del Ministerio de Salud, Trabajo y Bienestar de Japón para VYJUVEK®, marcando un hito importante como el primer medicamento genético aprobado en Japón para el tratamiento de la epidermólisis bullosa distrófica (DEB).
La aprobación permite opciones flexibles de administración, incluyendo la dosificación en el hogar y la administración por parte de los pacientes o familiares. VYJUVEK funciona entregando copias funcionales del gen humano COL7A1 para promover la cicatrización de heridas y la expresión sostenida de la proteína colágeno tipo VII funcional. La compañía espera lanzar el producto en Japón para finales de 2025, pendiente de la finalización de los procedimientos de reembolso.
Los datos clínicos que respaldan la aprobación incluyen resultados de un estudio de extensión abierto en Japón, donde los cuatro pacientes lograron el cierre completo de las heridas a los seis meses, consistente con los resultados previos del estudio de Fase 3 en EE. UU. El tratamiento ya ha recibido aprobaciones en Estados Unidos (mayo de 2023) y Europa (abril de 2025).
Krystal Biotech (NASDAQ: KRYS)는 일본 후생노동성으로부터 VYJUVEK®에 대한 판매 허가를 받았으며, 이는 일본에서 유전성 질환인 퇴행성 표피박리증(DEB) 치료를 위한 첫 번째 유전자 치료제 승인이라는 중요한 이정표입니다.
이번 승인은 유연한 투여 옵션을 허용하며, 환자나 가족이 집에서 직접 투여할 수 있습니다. VYJUVEK는 인간 COL7A1 유전자의 기능성 사본을 전달하여 상처 치유와 지속적인 기능성 제7형 콜라겐 단백질 발현을 제공합니다. 회사는 2025년 말까지 일본에서 출시할 예정이며, 보험 급여 절차 완료를 기다리고 있습니다.
승인을 뒷받침하는 임상 데이터는 일본에서 진행된 공개 라벨 확장 연구 결과를 포함하며, 네 명의 환자 모두 6개월 만에 완전 상처 폐쇄를 달성하여 미국 3상 연구 결과와 일치합니다. 이 치료제는 이미 미국(2023년 5월)과 유럽(2025년 4월)에서 승인을 받았습니다.
Krystal Biotech (NASDAQ : KRYS) a obtenu l'autorisation de mise sur le marché du ministère de la Santé, du Travail et du Bien-être du Japon pour VYJUVEK®, marquant une étape importante en tant que premier médicament génétique approuvé au Japon pour le traitement de l'épidermolyse bulleuse dystrophique (DEB).
Cette autorisation permet des options d'administration flexibles, y compris la posologie à domicile et l'administration par les patients ou leurs proches. VYJUVEK agit en délivrant des copies fonctionnelles du gène humain COL7A1 afin de favoriser la cicatrisation des plaies et l'expression soutenue de la protéine collagène de type VII fonctionnelle. L'entreprise prévoit de lancer le produit au Japon d'ici la fin 2025, sous réserve de l'achèvement des procédures de remboursement.
Les données cliniques soutenant cette approbation incluent les résultats d'une étude d'extension en ouvert menée au Japon, où les quatre patients ont obtenu une fermeture complète des plaies à six mois, conformément aux résultats précédents de l'étude de phase 3 aux États-Unis. Le traitement a déjà été approuvé aux États-Unis (mai 2023) et en Europe (avril 2025).
Krystal Biotech (NASDAQ: KRYS) hat von Japans Ministerium für Gesundheit, Arbeit und Wohlfahrt die Marktzulassung für VYJUVEK® erhalten, was einen bedeutenden Meilenstein darstellt als das erste in Japan zugelassene genetische Medikament zur Behandlung der dystrophischen Epidermolysis bullosa (DEB).
Die Zulassung erlaubt flexible Verabreichungsoptionen, einschließlich der Dosierung zu Hause durch Patienten oder Familienangehörige. VYJUVEK wirkt, indem es funktionale Kopien des menschlichen COL7A1-Gens liefert, um die Wundheilung und eine anhaltende Expression des funktionellen Kollagen-Typs VII zu gewährleisten. Das Unternehmen plant, bis Ende 2025 in Japan auf den Markt zu kommen, vorbehaltlich des Abschlusses der Erstattungsverfahren.
Die klinischen Daten zur Unterstützung der Zulassung stammen aus einer offenen Verlängerungsstudie in Japan, bei der alle vier Patienten nach sechs Monaten eine vollständige Wundheilung erreichten, was mit den Ergebnissen der US-amerikanischen Phase-3-Studie übereinstimmt. Die Behandlung wurde bereits in den USA (Mai 2023) und Europa (April 2025) zugelassen.
- First and only genetic medicine approved in Japan for DEB treatment
- Flexible administration options including home dosing
- 100% success rate in Japanese clinical trials with full wound closure at 6 months
- 10-year re-examination period granted in Japan
- Favorable safety profile demonstrated in clinical trials
- Launch timing dependent on pending reimbursement procedures
- Limited patient data from Japanese trials (only four patients)
Insights
VYJUVEK's approval in Japan gives Krystal Biotech its third major market for this first-in-class genetic therapy for DEB.
The approval of VYJUVEK by Japan's Ministry of Health, Labour and Welfare represents a significant regulatory milestone for Krystal Biotech, marking their third major market approval following previous authorizations in the US (May 2023) and Europe (April 2025). This approval has several notable elements that strengthen the company's market position:
The Japanese approval includes flexible administration options, allowing for home dosing and self-administration by patients or family members - a first for a genetic medicine in Japan. This patient-friendly approach eliminates the need for frequent hospital visits, potentially increasing therapy adoption and patient compliance.
Importantly, the approval comes with a 10-year re-examination period, providing substantial market protection in Japan. The regulatory pathway required special environmental safety confirmation under the Cartagena Act, demonstrating the product's favorable safety profile and minimal environmental dissemination risk.
The clinical validation included a successful open-label extension study in Japanese patients that mirrored positive outcomes from US trials, with all four completing patients achieving complete wound closure at six months. This consistency across different populations strengthens the therapy's credibility.
While timing for commercial availability depends on ongoing reimbursement procedures, the company expects to launch in Japan by year-end, potentially adding a new revenue stream within months. As the first and only genetic medicine approved in Japan for DEB, VYJUVEK addresses a significant unmet need for the hundreds of Japanese patients suffering from this debilitating genetic condition, targeting the root cause by delivering functional copies of the COL7A1 gene.
VYJUVEK approved for the treatment of DEB from birth with flexible administration options including home dosing and the option for administration by the patient or their family
VYJUVEK is the first and only genetic medicine approved in Japan for the treatment of DEB
PITTSBURGH, July 25, 2025 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS) announced today that on July 24, 2025, Japan’s Ministry of Health, Labour and Welfare (MHLW) granted marketing authorization to VYJUVEK® (beremagene geperpavec-svdt) for the treatment of wounds in patients with dystrophic epidermolysis bullosa (DEB), starting from birth. VYJUVEK is the first genetic medicine approved in Japan for the treatment of DEB and is designed to address the root cause of the disease by delivering functional copies of the human COL7A1 gene to provide wound healing and sustained functional type VII collagen protein expression with redosing. The Japanese approval allows for dosing at home or in a healthcare setting, with the option for administration by patients or their family members.
As per the approval issued by the MHLW, VYJUVEK is intended for use only in patients with a definite diagnosis of dystrophic epidermolysis bullosa. Genetic testing is not a requirement for treatment.
VYJUVEK is the first genetic medicine approved in Japan for home administration after undergoing regulatory evaluation and environmental safety confirmation for type 1 use in accordance with the Cartagena Act. Approval for home administration was granted based on VYJUVEK’s favorable safety profile and minimal risk of environmental dissemination when administered appropriately.
The timing for availability of VYJUVEK in Japan will depend on completion of reimbursement procedures currently underway. The Company currently expects to launch in Japan by the end of this year.
“For far too long, DEB patients in Japan have suffered from a lack of corrective therapies to manage their complex and often debilitating disease,” said Hiroshi Kasamoto, General Manager of Japan at Krystal Biotech. “The approval of VYJUVEK in Japan is a long-awaited and urgently needed breakthrough, offering the potential to dramatically improve the lives of DEB patients across the country. With this important milestone, we remain on track to commercially launch VYJUVEK in Japan later this year and bring this innovative therapy to the many hundreds of DEB patients in need.”
The approval by Japan’s MHLW was based on a comprehensive clinical dataset, which included results from an open label extension study conducted in Japanese patients. Results from the Japanese open label extension study closely mirrored prior clinical experience, including the Company’s Phase 3 study in the United States, with all four patients that completed the study achieving the primary study endpoint of full wound closure at six months. VYJUVEK was also well tolerated and exhibited a safety profile in Japanese patients that was consistent with previous studies.
“The approval of VYJUVEK in Japan is another important milestone for our organization and the DEB patients we serve,” said Suma Krishnan, President of Research and Development at Krystal Biotech. “With recent approvals now in both Japan and Europe, we are making tremendous progress towards our goal of improving outcomes for DEB patients around the world.”
The re-examination period for VYJUVEK in Japan is ten years.
VYJUVEK was previously approved by the Food and Drug Administration in the United States in May 2023 and by the European Commission in April 2025.
JAPAN INDICATION
Dystrophic epidermolysis bullosa
About Krystal Biotech, Inc.
Krystal Biotech, Inc. (NASDAQ: KRYS) is a fully integrated, commercial-stage, global biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs. VYJUVEK®, the Company’s first commercial product, is the first-ever redosable gene therapy, and the first genetic medicine approved in the United States, Europe, and Japan 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).
About Cartagena Act
The Act on the Conservation and Sustainable Use of Biological Diversity through Regulations on the Use of Living Modified Organisms, also referred to as the Cartagena Act, is a law regulating the use of living and/or genetically modified organisms in Japan, including the use of such organisms as medical products.
Forward-Looking Statements
Any statements in this press release about future expectations, plans and prospects, including statements about the Company’s current expectation of launching VYJUVEK in Japan by the end of 2025; 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 related to reimbursement for VYJUVEK; and such 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. 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
What is the significance of VYJUVEK's approval in Japan for Krystal Biotech (NASDAQ: KRYS)?
How does VYJUVEK work in treating DEB patients?
What were the results of VYJUVEK's clinical trials in Japan?
When will VYJUVEK be available for patients in Japan?
How can VYJUVEK be administered to DEB patients in Japan?
