Krystal Biotech has received approval from Japan’s Ministry of Health, Labour and Welfare (MHLW) for its gene therapy, Vyjuvek, to treat dystrophic epidermolysis bullosa (DEB).
Vyjuvek, which targets the root cause of DEB by delivering functional copies of the COL7A1 gene, can be administered at home or in healthcare settings.
The therapy had previously gained approval from the US Food and Drug Administration (FDA) in May 2023 and the European Commission (EC) in April 2025.
Vyjuvek is the first genetic medicine in Japan approved for home administration after regulatory evaluation under the Cartagena Act.
It is the first redosable gene therapy approved in Japan, Europe, and the US for this condition.
Krystal Biotech Japan general manager Hiroshi Kasamoto said: “For far too long, DEB patients in Japan have suffered from a lack of corrective therapies to manage their complex and often debilitating disease.
“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 MHLW approval follows a favourable safety profile and minimal environmental risk, confirmed through complete clinical data, including a study conducted in Japanese patients.
The Japanese approval does not require genetic testing for treatment, but it is indicated for patients with a confirmed diagnosis of DEB.
The decision was supported by data showing full wound closure at six months in all four patients in a Japanese open-label extension study.
The US-based biotechnology company anticipates launching Vyjuvek in Japan by the end of the year, pending the completion of reimbursement procedures.
Krystal Biotech research and development president Suma Krishnan said: “The approval of Vyjuvek in Japan is another important milestone for our organisation and the DEB patients we serve.
“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.”
