Krystal Biotech touts topical gene therapy uptake, plots overseas launches

After a 2023 approval from the FDA, Krystal Biotech has collected more than $95 million from its launch of Vyjuvek, the first treatment for the rare skin disease dystrophic epidermolysis bullosa (DEB). Now, the company has sights set on two other major markets to support its blockbuster ambitions.

With first-quarter sales of $45.3 million, Vyjuvek’s performance since its May 2023 FDA nod is in line with the "top tier” of recent rare disease launches, CEO Krish Krishnan said on the company’s first-quarter earnings conference call.

Krystal has previously detailed a $1 billion global market opportunity for the product. One upcoming checkpoint on the road would be overseas approvals.

Vyjuvek is already under review in Europe, with a launch in Germany expected late this year or early next, Krishnan said on the call. Krystal looks to file for approval in Japan during the second half of this year and is currently building out its global infrastructure to support more launches.

Other than its status as the first DEB treatment, Vyjuvek holds the title of the first FDA-approved topical gene therapy. When directly applied to DEB wounds, the med essentially binds the skin together by delivering copies of the COL7A1 gene. Patients with the disease live with extremely fragile skin that tears with the slightest friction, causing open wounds that can lead to serious outcomes.

The topical gel is administered weekly. While a frequent dosing schedule often poses a barrier to uptake, patient compliance for Vyjuvek is “stellar,” Krystal’s head of patient access, analytics and operations, Jennifer McDonough, said on the company’s first-quarter call.

More than 90% of patients on Vyjuvek are applying the med on a weekly basis, Krishnan explained. That means just a “handful” of patients may miss a dose every once in a while, but “most of the patients” remain on track.

The compliance levels are buoyed by the drug's status as an at-home treatment, which Krishnan previously called “one of the best things we got on the label.” Nearly all weekly treatments are occuring in patients' homes, as Vyjuvek administration can easily fit into preexisting wound care routines, Krystal's head of U.S. sales and marketing, Christine Wilson, pointed out on the earnings call.

To capture further growth, the company is utilizing targeted social media ads as well as educational programs to drive adoption and build on a prescriber base that currently numbers "in the hundreds,” Wilson said. Positive patient experiences and increasing familiarity should help establish Vyjuvek as a DEB standard of care in the upcoming years, the executive added.

For this year, analysts with Evercore ISI estimate the med's sales will reach $285 million based on a “strong anticipated trajectory," the team wrote in a note to clients.