Krystal Biotech Sells Rare Pediatric Disease Priority Review Voucher For $100M

Krystal Biotech, Inc. KRYS has completed the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for $100 million.

The company was awarded the PRV following the U.S. Food and Drug Administration (FDA) accelerated approval of Vyjuvek (beremagene geperpavec-svdt).

Under the Rare Pediatric Disease PRV Program, the FDA awards priority review vouchers to sponsors of rare pediatric disease product applications that meet certain criteria. 

Vyjuvek treats patients six months or older with a skin blistering disorder or dystrophic epidermolysis bullosa (DEB). The FDA approved it on May 2023.

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

Read More: Unlocking a $750 Million Opportunity: Krystal Biotech's Vyjuvek and its Potent Impact on Dystrophic Epidermolysis Bullosa Treatment

Krystal estimates about 3,000 total DEB patients in the U.S, split roughly 50/50 between recessive and dominant forms. Of those patients, approximately 1,100 are diagnosed or identified via claims analytics, and these are likely more severe. 

Price Action: KRYS shares are trading higher by 0.15% to $119.58 on the last check Monday.