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 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.

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.