FDA Approves First Redosable Gene Therapy, Krystal Biotech's Treatment For Skin Blistering Disorder

The FDA approved Krystal Biotech Inc's KRYS Vyjuvek (beremagene geperpavec-svdt) for the treatment of patients six months of age or older with dystrophic epidermolysis bullosa (DEB). 

Epidermolysis bullosa cause the skin to be very fragile and to blister easily.

Vyjuvek is designed to address the genetic root cause of DEB by delivering functional copies of the human COL7A1 gene to provide wound healing and sustained functional COL7 protein expression with redosing. 

Vyjuvek is the first-ever redosable gene therapy and the first and only medicine approved by the FDA for DEB, both recessive and dominant, that can be administered by a healthcare professional.

Vyjuvek is expected to be available in the U.S. in the third quarter of 2023, and the company will begin the promotion of Vyjuvek immediately.

With this approval, the FDA issued the Company a Rare Pediatric Disease Priority Review Voucher, which confers priority review to a subsequent drug application that would not otherwise qualify for priority review. 

The company anticipates starting the official Marketing Authorization Application procedure in the second half of 2023, with potential approval in 2024. The company is also working with Japan's Pharmaceuticals and Medical Devices Agency to study Vyjuvek and seek approval for a potential launch in 2025.

Krystal Biotech announced a private placement of 1.73 million at $92.50 for gross proceeds of approximately $160 million.

Price Action: KRYS shares are down 6.31% at $90 on the last check Monday.