Krystal Biotech (KRYS) Announces FDA Accepts its Biologics License Application for Dystrophic Epidermolysis Bullosa

Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), the leader in redosable gene therapy, today announced that the US Food and Drug Administration (FDA) has accepted for filing the Company’s Biologics License Application (BLA) for B-VEC for the treatment of patients with dystrophic epidermolysis bullosa (DEB). The application has been granted Priority Review designation, and the Prescription Drug User Fee Act (PDUFA) action date is February 17, 2023. The FDA stated that it is not currently planning to hold an advisory committee meeting to discuss the application.

"We are delighted to receive the FDA’s acceptance of our BLA submission and move one step closer to potentially bringing a medicine to fundamentally treat DEB patients,” said Suma Krishnan, Co-Founder and President, Research & Development of Krystal Biotech. “We are committed to working closely with the FDA to bring this potential, first-ever treatment to patients living with DEB as quickly as possible.”

The BLA submission for B-VEC is supported by data from two placebo controlled clinical trials - the GEM-3 trial (NCT04491604) and the GEM-1/2 trial (NCT03536143).

Priority Review designation is granted to applications for medicines that treat a serious condition, and if approved, would provide a significant improvement in the safety or effectiveness of the treatment, prevention, or diagnosis of a serious condition. B-VEC was previously granted Fast Track designation and Regenerative Medicine Advanced Therapy (RMAT) by the FDA for the treatment of DEB.

The Company plans to submit a marketing authorization application to the European Medicines Agency (EMA) in the second half of 2022.

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