US FDA Approves Gene Therapy for “Butterfly Children”

The US Food and Drug Administration (FDA) has approved Krystal Biotech’s Vyjuvek, the first gene therapy for dystrophic epidermolyis bullosa (DEB), a rare disease that leaves affected patients, dubbed “butterfly children” with skin that damages extremely easily and heals poorly. The treatment, which would cost $24,250 per dose, is applied to wounded skin, delivering a collogen gene that is missing in patients.

According to Ryan Cross, “In clinical trials, a repeated application of the therapy to a patient’s raw wounds help them heal faster and stay healed longer, at least for a few months. The treatment is not a cure, and it only works on wounded skin, but doctors and patient advocates alike are relieved to at last have something to help patients cope with the disease.”

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(Source: Endpoints News, May 19^th, 2023)