FDA approves groundbreaking rub-on gene therapy for rare skin disorder.
TL;DR Summary
The US FDA has approved Krystal Biotech's Vyjuvek, a topical gene therapy for patients with dystrophic epidermolysis bullosa, a rare genetic skin disorder. The therapy is expected to be available in the US in Q3 2023 and is Krystal's first to be approved in the country. The FDA approval is supported by data from early-to-mid-stage and late-stage studies, which showed Vyjuvek completely healed wounds in about 65% of the participants.
Topics:business#dystrophic-epidermolysis-bullosa#fda#gene-therapy#healthcare#krystal-biotech#vyjuvek
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