Patients with the rare dystrophic epidermolysis bullosa disorder suffer from open wounds, causing skin infections and are at an increased risk of vision loss, scarring and skin cancer. Most patients rarely survive beyond 30 years of age.
The therapy, Vyjuvek, is expected to be available in the United States in the third quarter of 2023, Krystal Biotech CEO Krish Krishnan told Reuters ahead of the FDA decision.
"We have been preparing for a commercial launch for the last 18 months if not longer. Our intent is to provide access to all the patients if possible in the United States," Krishnan said.
The therapy, which has been approved for patients aged six months or older with either recessive or dominant forms of the disorder, is Krystal's first to be approved in the United States.
About 9,000 to 10,000 people suffer from dystrophic epidermolysis bullosa globally, including a U.S. population of about 3,000 patients, according to Krystal Biotech.
The company did not immediately respond to a Reuters request for comment on the therapy's price.
The FDA approval is supported by data from an early-to-mid-stage study, and a late-stage study of 31 patients which showed Vyjuvek completely healed wounds in about 65% of the participants, compared with just 21.6% of the patients on placebo.
(Reporting by Pratik Jain in Bengaluru; Editing by Shailesh Kuber and Vinay Dwivedi)
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