VYJUVEK® Receives Groundbreaking Approval for DEB Treatment
VYJUVEK® Approved for Dystrophic Epidermolysis Bullosa Treatment
VYJUVEK approved for DEB treatment from birth, offering flexible home administration options for patients and their families.
This breakthrough marks VYJUVEK as the first genetic medicine approved in Japan for DEB.
Recently, Krystal Biotech, Inc. (NASDAQ: KRYS) announced that its innovative treatment, VYJUVEK® (beremagene geperpavec-svdt), has gained marketing authorization from Japan's Ministry of Health, Labour and Welfare for patients with dystrophic epidermolysis bullosa (DEB), beginning from birth. This significant achievement allows for treatment versatility, ensuring patients receive care either at home or in a professional healthcare setting. Notably, family members can also administer the treatment, thereby enhancing accessibility for patients.
VYJUVEK operates by addressing the fundamental cause of DEB through the delivery of functional copies of the human COL7A1 gene. This delivery promotes wound healing and supports a sustained expression of type VII collagen protein through a redosing mechanism. It effectively opens the door for patients, who have long awaited effective therapies for their condition.
Additionally, the approval by Japan's MHLW is aimed specifically at patients with a confirmed diagnosis of DEB, simplifying the process as genetic testing is not mandatory for initiating treatment.
Significance of the Approval
A landmark in genetic medicine, VYJUVEK is the first product of its kind approved for home usage in Japan, following thorough regulatory evaluations that ensured environmental safety and compliance with the Cartagena Act. Its safety profile demonstrates a minimal risk of environmental dissemination when utilized correctly, accentuating its promise as a safe home treatment option.
The timing for VYJUVEK's launch in Japan will rely heavily on the ongoing reimbursement procedures, but the company anticipates that it will become available by the year's end. This prospect excites many within the company and the medical community, who recognize the urgent need for effective solutions for DEB patients.
According to Hiroshi Kasamoto, General Manager of Japan at Krystal Biotech, "For far too long, DEB patients have suffered from a lack of effective therapies. The approval of VYJUVEK is a significant breakthrough, potentially revolutionizing the lives of many DEB patients in Japan. We look forward to launching VYJUVEK and making a meaningful difference in the lives of those affected."
The approval is grounded in a robust clinical dataset, highlighting results from a Japanese open-label extension study. Outcomes from this study closely paralleled previous clinical trials, including the successful Phase 3 study conducted in the U.S., wherein all four participants achieved the primary endpoint of complete wound closure at six months. VYJUVEK's safety profile continued to show compatibility with earlier research findings.
The Road Ahead for Krystal Biotech
Suma Krishnan, President of Research and Development, noted, "The approval of VYJUVEK in Japan is a crucial milestone for our organization. With recent approvals in both Japan and Europe, we are making great strides toward enhancing outcomes for DEB patients globally." This sentiment reflects a promising trajectory for Krystal Biotech as they aim to extend their innovative treatments to broader markets.
The re-examination period for VYJUVEK in Japan is set for ten years, providing a structured framework for ongoing evaluation and adaptation based on patient outcomes and treatment efficacy. Following its successful FDA approval in the U.S. back in May 2023 and the European Commission's approval in April 2025, the momentum for VYJUVEK continues to build.
Indication in Japan
Dystrophic epidermolysis bullosa.
About Krystal Biotech, Inc.
Krystal Biotech, Inc. (NASDAQ: KRYS) is a fully integrated biotechnology company committed to discovering and commercializing genetic therapies for diseases with significant unmet medical needs. VYJUVEK® is their flagship redosable gene therapy, representing a major advance in the fight against DEB. The company's pipeline includes promising genetic medicines targeting various fields, including respiratory illnesses, oncology, dermatology, and aesthetics, showcasing their dedication to combating numerous health challenges. Krystal Biotech is headquartered in Pittsburgh, Pennsylvania. For more information, visit the company's website and connect with them on LinkedIn and X.
Frequently Asked Questions
What is VYJUVEK® used for?
VYJUVEK® is approved for the treatment of dystrophic epidermolysis bullosa (DEB) and is the first genetic medicine authorized in Japan for this condition.
Who can administer VYJUVEK?
The treatment can be administered at home or healthcare facilities, with options for patients or family members to provide care.
Is genetic testing required for VYJUVEK® treatment?
No, genetic testing is not mandatory for patients seeking treatment with VYJUVEK®.
When is VYJUVEK expected to be available in Japan?
The launch is anticipated by the end of this year, pending the completion of reimbursement procedures.
What is the significance of VYJUVEK's approval for DEB patients?
The approval represents a monumental advancement in therapeutic options for DEB patients, potentially drastically improving their quality of life and providing effective care.
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