Pittsburgh-based Krystal Biotech Inc., a biotechnology company focused on genetic medicines to treat diseases with high unmet medical needs, recently completed the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for $100 million.
The U.S. Food and Drug Administration (FDA), under the Rare Pediatric Disease Priority Review Voucher Program, awards priority review vouchers to sponsors of rare pediatric disease product applications that meet certain criteria. The program encourages the development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.
PRVs can be sold, transferred or redeemed to receive priority review of a marketing application for a different product.
The FDA awarded Krystal Biotech the PRV following the accelerated approval of the drug VYJUVEK for the treatment of recessive or dominant dystrophic epidermolysis bullosa for patients older than six 6 months old.
VYJUVEK is the company’s first commercial product. It is the first redosable gene therapy, and the only pharmaceutical the FDA has approved for the treatment of dystrophic epidermolysis bullosa.
Krystal Biotech currently is advancing a preclinical and clinical pipeline of investigational genetic medicines in aesthetics, dermatology, oncology, ophthalmology, and respiratory.
The company focuses on the discovery, development and commercialization stages of genetic medicines.