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CBER chief Peter Marks to consider clinical hold options as they ‘set investors’ jitters’ – Endpoints News
Children born with a rare disease that makes their skin as fragile as a butterfly’s wings, leaving them vulnerable to frequent sores and constant pain, will finally have access to a new treatment that aims to fix the genetic cause of the condition.
The therapy, known as Vyjuvek and made by Krystal Biotech, was approved by the FDA on Friday afternoon to treat patients six months or older with dystrophic epidermolysis bullosa, or DEB. The drug is a long-awaited solution for many families affected by the hellish condition, for which there are no approved treatments in the United States.
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