FDA approves first gene therapy in a moisturizer bottle

What's the news: The FDA just approved the first-ever topical gene therapy.

Why should we believe it: This news was announced on the FDA site two weeks ago. The FDA approval came thanks to a study published last December, which provided proof for the efficacy of this new topical treatment, which is called Vyjuvek.

So what is Vyjuvek? The therapy targets a collagen gene mutation that causes a rare and painful skin disease, dystrophic epidermolysis bullosa (DEB). Imagine constant, painful, peeling sunburns, over much of your body — that gives you an idea of DEB.

DEB is caused by a single gene mutation, which makes it a perfect candidate for gene therapy. The new thing here is that, unlike other gene therapies, which have to be injected, ingested, or otherwise inserted into the body, Vyjuvek is simply applied to the skin. The therapy is 2.5x more effective that placebo, and closed 66% of DEB skin wounds.

Why this is a big deal: DEB is a rare disease, but one that causes significant misery for people who have it. Vyjuvek is not a cure for DEB, but is a significant new treatment. Brett Kopelan, Executive Director of debra, an organization that supports people with DEB, called Vyjuvek a "monumental milestone in the treatment of this horrible disorder."

Beyond being good news for patients with DEB, this FDA approval offers hope for future topical gene therapies that deal with other skin disorders, and perhaps, simply with regular aging. Not surprisingly, Krystal Biotech, the company that produces Vyjuvek, has already spun off a new company, Jeune, to focus on cosmetic applications.