With global expansion planned, Krystal lays out blockbuster ambitions for topical gene therapy

Not even one year after becoming a commercial biotech, Krystal Biotech has blockbuster dreams for its flagship product. 

The company launched its topical gene therapy Vyjuvek six months ago and has so far collected $50.7 million in product revenues. The performance is “tracking closely” to "the best recent rare disease launches,” CEO Krish Krishnan said in the company’s fourth-quarter and full-year earnings release.

Krystal's Vyjuvek made history in more ways than one as the first med approved to treat rare skin disease dystrophic epidermolysis bullosa (DEB) and the first topical gene therapy to cross the FDA finish line in May 2023.

The topical gel is a genetically modified herpes simplex type 1 virus that delivers copies of the COL7A1 gene when directly applied to DEB wounds. Patients with DEB have extremely fragile skin that rips and blisters with the slightest friction, causing open wounds that are prone to skin infections and fibrosis.

Importantly, Vyjuvek can be administered at home, which is “one of the best things we got on the label,” Krishnan said on the company's earnings conference call Monday. “Compliance is high because of this.”

So far, 35% of the 1,200 eligible DEB patients in the U.S. have initiated start forms, with 228 reimbursement approvals being granted to date, according to the company.

Currently, it takes about 30 days to start on the drug after completing the start form, but the company hopes to shrink that period by “maybe two to three weeks” by the second half of this year, Krishnan said.

Krsytal isn’t yet done in DEB, which it is committed to “treating comprehensively,” R&D head Suma Krishnan said on the company’s earnings conference call. Its next endeavor is an eye-drop formulation of Vyjuvek to treat ocular DEB complications, which occur in more than 25% of DEB patients.

Even without the eye-drop formulation, Krystal is “very definitive” on the $1 billion market opportunity it sees for the product. Those hopes are bolstered by feedback from regulatory authorities in Europe and Japan, both regions in which the company hopes to launch by 2025.

Still, an eye-drop form and ocular label expansion could add some $250 million to $350 million in potential sales, Krishnan said in a “very premature” estimate.

Vyjuvek’s approval ushered in a “whole new paradigm” to treat genetic diseases, Krishnan said at the time. With the approval in hand, Krystal is also testing pipeline candidates in cystic fibrosis, solid tumors and other diseases. The company has a total of five clinical trials planned for this year.