Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), the leader in redosable gene therapy, announced today that the European Medicines Agency (EMA) Pediatric Committee (PDCO) has adopted a positive opinion on the Pediatric Investigation Plan (PIP) for beremagene geperpavec (B-VEC) for the treatment of dystrophic epidermolysis bullosa.
As part of the regulatory process for the registration of new medicines in Europe, the EMA requires companies to provide a PIP outlining their strategy for investigation of the new medicinal product in the pediatric population. An approved PIP is a prerequisite for filing a Marketing Authorization Application (MAA). The positive opinion is based on the B-VEC non-clinical safety program as well as data from the clinical studies conducted in the US that will be included in the upcoming MAA.
“The approval of the PIP provides a clear path forward, and we look forward to working with the EMA and PDCO to bring this important treatment to the European market as soon as possible,” said Suma Krishnan, President, Research & Development.
Based on this positive opinion, the Company would be eligible for up to an additional two years of marketing exclusivity in the EU, on top of the ten-year EU market exclusivity after market approval in the EU.
The U.S. Food and Drug Administration (FDA) and EMA have each granted B-VEC orphan drug designation for the treatment of DEB. The FDA has granted B-VEC fast track designation, Regenerative Medicine Advanced Therapy (RMAT) and rare pediatric disease designation for the treatment of DEB. B-VEC is eligible to receive a Priority Review Voucher (PRV) following approval of B-VEC in the US. The EMA granted PRIority MEdicines (PRIME) eligibility for B-VEC to treat DEB.
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.