Krystal Biotech Provides Update on EMA’s Ongoing Regulatory Review of B-VEC for Dystrophic Epidermolysis Bullosa
Krystal Biotech (NASDAQ: KRYS) provided an update on the European Medicines Agency's (EMA) review of B-VEC for dystrophic epidermolysis bullosa (DEB). The EMA's Committee for Medicinal Products for Human Use (CHMP) cancelled the scheduled Oral Explanation and requested written responses to remaining issues. Notably, there are no Major Objections outstanding regarding B-VEC's full approval. The company now expects a CHMP opinion in 1Q 2025, while maintaining its planned commercial launch in Germany for Q2 2025.
Krystal Biotech (NASDAQ: KRYS) ha fornito un aggiornamento sulla revisione del B-VEC da parte dell'Agenzia Europea per i Medicinali (EMA) per la distrofica epidermolisi bollosa (DEB). Il Comitato per i Prodotti Medicinali per Uso Umano (CHMP) dell'EMA ha annullato la spiegazione orale programmata e ha richiesto risposte scritte alle questioni rimanenti. È importante notare che non ci sono obiezioni significative in sospeso riguardo all'approvazione completa del B-VEC. L'azienda ora prevede un parere del CHMP nel 1Q 2025, mentre mantiene il lancio commerciale programmato in Germania per il Q2 2025.
Krystal Biotech (NASDAQ: KRYS) proporcionó una actualización sobre la revisión del B-VEC por parte de la Agencia Europea de Medicamentos (EMA) para la epidermólisis bullosa distrófica (DEB). El Comité de Productos Medicinales para Uso Humano (CHMP) de la EMA canceló la explicación oral programada y solicitó respuestas por escrito a las cuestiones restantes. Es importante señalar que no hay objeciones importantes pendientes con respecto a la aprobación total de B-VEC. La empresa ahora espera un dictamen del CHMP en el 1Q 2025, mientras mantiene su lanzamiento comercial planificado en Alemania para el Q2 2025.
크리스탈 바이오텍 (NASDAQ: KRYS)은 진피형 수포성 피부염 (DEB)에 대한 B-VEC의 유럽 의약품청(EMA) 검토 업데이트를 제공했습니다. EMA의 인간 사용 의약품 위원회(CHMP)는 예정된 구두 설명을 취소하고 남은 문제에 대한 서면 답변을 요청했습니다. 특히, B-VEC의 전체 승인을 위한 주요 이의는 없다는 점이 주목할 만합니다. 회사는 2025년 1분기에 CHMP의 의견을 기대하고 있으며, 2025년 2분기에 독일에서의 상업적 출시를 계획하고 있습니다.
Krystal Biotech (NASDAQ: KRYS) a fourni une mise à jour sur l'examen du B-VEC par l'Agence européenne des médicaments (EMA) pour la dystrophie épidermolyse bulleuse (DEB). Le Comité des médicaments à usage humain (CHMP) de l'EMA a annulé l'explication orale prévue et a demandé des réponses écrites aux questions restantes. Il est à noter qu'il n'y a pas d'objections majeures en suspens concernant l'approbation complète du B-VEC. L'entreprise s'attend désormais à un avis du CHMP au 1er trimestre 2025 tout en maintenant son lancement commercial prévu en Allemagne pour le 2e trimestre 2025.
Krystal Biotech (NASDAQ: KRYS) hat ein Update zur Überprüfung von B-VEC durch die Europäische Arzneimittelbehörde (EMA) für dystrophische epidermolysis bullosa (DEB) bereitgestellt. Der Ausschuss für Humanarzneimittel (CHMP) der EMA hat die geplante mündliche Erklärung abgesagt und um schriftliche Antworten zu den verbleibenden Fragen gebeten. Es ist bemerkenswert, dass keine wesentlichen Einwände gegen die vollständige Genehmigung von B-VEC bestehen. Das Unternehmen erwartet nun eine CHMP-Stellungnahme im 1. Quartal 2025 und plant, im 2. Quartal 2025 in Deutschland kommerziell zu lancieren.
- No Major Objections outstanding from EMA for B-VEC's full approval
- German market launch timeline remains on track for Q2 2025
- CHMP opinion delayed to 1Q 2025 due to additional documentation requirements
- Cancellation of scheduled Oral Explanation meeting
Insights
The EMA review delay for B-VEC, while pushing the CHMP opinion to Q1 2025, appears to be a procedural matter rather than a significant setback. The absence of Major Objections is particularly encouraging, suggesting that the core efficacy and safety profile of B-VEC for DEB treatment meets regulatory standards. The maintained Q2 2025 launch timeline for Germany, Europe's largest pharmaceutical market, indicates minimal commercial impact.
The focus on post-marketing requirements rather than major clinical concerns suggests a robust regulatory package. For rare diseases like DEB, which has treatment options, regulatory authorities often work collaboratively with companies to ensure proper post-market monitoring while maintaining access to innovative therapies. This approach aligns with standard practice for breakthrough therapies in rare diseases.
The regulatory update presents a neutral market impact. While the CHMP opinion delay might initially appear concerning, several key factors mitigate potential negative implications:
- Commercial launch timeline in Germany remains unchanged
- No Major Objections from EMA indicates high likelihood of approval
- Focus on post-marketing issues rather than efficacy/safety concerns
The maintained launch schedule for Germany is particularly significant as it represents the primary European market entry point. The company's confident stance on addressing remaining issues, coupled with the preservation of key commercial timelines, suggests minimal impact on projected European revenue streams. This regulatory progression appears to be following a typical path for innovative rare disease therapies.
CHMP opinion now expected in 1Q 2025
No Major Objections outstanding; continue to expect Germany launch in 2Q 2025
PITTSBURGH, Dec. 09, 2024 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company, today announced that the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) cancelled the Oral Explanation regarding the Company’s Marketing Authorization Application for beremagene geperpavec-svdt (B-VEC) for the treatment of dystrophic epidermolysis bullosa (DEB) that was scheduled for December 6, 2024, and asked the Company to submit written responses to the remaining outstanding issues.
There are no Major Objections outstanding from the EMA with respect to the full approval of B-VEC.
“We are confident in our ability to address the remaining post-marketing issues, and we believe that this additional exchange with EMA will ultimately maximize benefit and convenience to patients suffering from DEB,” said Suma Krishnan, President of Research and Development at Krystal Biotech.
The Company now anticipates a CHMP opinion in 1Q 2025 while the launch timelines remain unchanged with a commercial launch in Germany still planned for Q2 2025.
About Dystrophic Epidermolysis Bullosa (DEB)
DEB is a rare and severe disease that affects the skin and mucosal tissues. It is caused by one or more mutations in a gene called COL7A1, which is responsible for the production of the protein type VII collagen (COL7) that forms anchoring fibrils that bind the dermis (inner layer of the skin) to the epidermis (outer layer of the skin). The lack of functional anchoring fibrils in DEB patients leads to extremely fragile skin that blisters and tears from minor friction or trauma. DEB patients suffer from open wounds, which leads to skin infections, fibrosis which can cause fusion of fingers and toes, and ultimately an increased risk of developing an aggressive form of squamous cell carcinoma which, in severe cases, can be fatal.
About B-VEC and VYJUVEK
B-VEC is a non-invasive, redosable gene therapy built to deliver two copies of the COL7A1 gene to treat DEB at the molecular level by providing the patient’s cells the template to make normal COL7 protein, thereby addressing the fundamental disease-causing mechanism. B-VEC was approved by U.S. Food and Drug Agency (FDA) in May 2023 for the treatment of DEB and is marketed and sold in the U.S. under the name VYJUVEK®. For more information on VYJUVEK, 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
This press release contains forward-looking statements, including those regarding the EMA’s review timeline and approval prospects for B-VEC, and the Company’s plans and timeline for its commercial launch of B-VEC in the European Union. Actual outcomes may differ materially based on various factors, including uncertainties associated with regulatory review and marketing approvals 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. 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 is Krystal Biotech (KRYS) expecting EMA's CHMP opinion for B-VEC?
When will Krystal Biotech (KRYS) launch B-VEC in Germany?
Are there any Major Objections from EMA regarding Krystal Biotech's (KRYS) B-VEC approval?
