FDA approves 1st drug for rare skin condition

The FDA on May 19 approved the first drug to treat patients with dystrophic epidermolysis bullosa — a rare genetic disorder that causes fragile skin and persistent sores. 

The drug approval was for Vyjuvek, made by Krystal Biotech. It is a topical gene therapy meant to treat wounds in patients 6 months and older and works by delivering normal copies of a type of collagen protein known as COL7 to the skin, enabling it to heal, according to an FDA news release. The most common adverse reactions to the treatment include itching, chills, redness, rash, cough and runny nose. 

"Vyjuvek is a genetically modified (engineered in a laboratory) herpes-simplex virus used to deliver normal copies of the COL7A1 gene to the wounds," the FDA said in a statement. "COL7 molecules arrange themselves into long, thin bundles that form anchoring fibrils that hold the epidermis (skin) and dermis together, which is essential for maintaining the integrity of the skin."

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