A South Side-based biotech company has reached a major drug-approving milestone for a topical product that aims to treat rare medical conditions that lead to easy skin blistering.

Krystal Biotech Inc. (NASDAQ: KRYS) announced that the U.S. Food and Drug Administration approved the use of VYJUVEK to treat dystrophic epidermolysis bullosa (DEB) for patients who are six months of age or older.

The company said VYJUVEK is the first and only drug approved by the FDA to treat DEB in its recessive and dominant forms. Health care officials are able to administer the drug in a medical setting or at a patient's home.

"VYJUVEK both heals patient wounds and prevents skin from re-blistering because it actually corrects the underlying skin defect of [DEB]," Dr. M. Peter Marinkovich, primary investigator of the GEM-3 trial, Director of the Blistering Disease Clinic at Stanford Health Care and associate professor of dermatology at the Stanford University School of Medicine, said in a prepared statement. "Because it’s safe and easy to apply directly to wounds, it doesn't require a lot of supporting technology or specialized expertise, making VYJUVEK highly accessible even to patients who live far away from specialized center."

Approval from the FDA comes after Krystal Biotech completed two prior clinical studies on the drug.

"For so many years, all we have been able to offer DEB patients was palliative care, but now, based on the strength of the company’s clinical trial data, there is a safe and effective FDA-approved treatment," Suma Krishnan, president of research & development at Krystal Biotech, said in a prepared statement.

Added Krish S. Krishnan, chairman and CEO of Krystal Biotech, in a statement: "Today’s landmark approval of VYJUVEK as the first redosable gene therapy ushers in a whole new paradigm to treat genetic diseases and is an important milestone for patients affected by DEB as well as their families and caregivers. … For Krystal, this is a transformative achievement that highlights our commitment to developing and commercializing novel therapies for patients with rare diseases and demonstrates Krystal’s capability as a fully-integrated company ready to launch and bring VYJUVEK to patients as quickly as possible and deliver additional transformative medicines to patients as we advance our pipeline."

Matt Smith, chief growth officer at the Allegheny Conference on Community Development, applauded the achievement.

"FDA approval for Krystal Biotech’s revolutionary gene therapy for dystrophic epidermolysis bullosa (DEB) is an incredible milestone. We couldn’t be prouder that this company – which started, scaled and succeeded in the Pittsburgh region – has attained such a medical breakthrough and coveted achievement," Smith said in a statement. "Krystal Biotech’s concept-to-commercialization success in developing the first and only FDA-approved medication to treat DEB is proof of what is possible in Pittsburgh. Why it’s happening here is largely due to the comprehensive efforts in motion to ensure that companies committed to saving lives and advancing wellness find the talent, space and community of support in our region that let them think big, solve big problems and thrive."

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Nate Doughty

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