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

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

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.

At a vial price of $24,250 WAC, the expected annual steady-state pricing per patient is $631,000, or $485,000 annually, after mandatory government discounts, above its prior estimate for steady-state usage of $450,000.

The analysts also highlight one question for investors regarding the addressable market size given the rare nature of DEB, and the early launch metrics over the coming quarters will be essential to help investors determine the ultimate size of the addressable market. 

Krystal estimates about 3,000 total DEB patients in the United States, 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. 

William Blair maintains Outperform rating and says the approval is transformational for Krystal. It estimates worldwide sales for Vyjuvek of $1.1 billion in 2030, $668 million of which are U.S. sales with a penetration in the U.S. of 75% of eligible patients with DEB. 

HC Wainwright says that the approval of Vyjuvek provides validation for Krystal's gene therapy platform and a positive readthrough to its growing pipeline. It reiterates a Buy rating with a price target of $139 from $119.

With a company estimated $750 million market opportunity, HC Wainwright analyst projects $900 million in U.S. peak sales.

The analyst projects a significant cash inflow from a potential sale of Vyjuvek's Priority Review voucher, which could garner an average of $75-$110 million based on recent PRV sales.

Price Action: KRYS shares are up 22.40% at $117.53 on Monday.