I'm an independent healthcare analyst with more than 24 years of experience analyzing healthcare and pharmaceuticals.
The promise of gene therapy is to cure diseases associated with faulty or missing genes. Yet the high upfront costs, uncertainty surrounding long-term durability and adverse events in some patients have often impeded market uptake.late last month said it expects a “very strong product launch” of a recently approved gene therapy made by Krystal Biotech, the topical gel Vyjuvek indicated for a rare skin disease, it caught people’s attention.
But Vyjuvek is different. Unlike the vast majority of current gene therapies on the market today, it’s not meant to be a one-off cure. Vyjuvek is the first and only Food and Drug Administration-approved topical gene therapy that can be re-dosed. It is also the first gene therapy to be applied topically to the skin, rather than infused.. DEB is a condition that causes the skin to be extremely fragile and easily blister which can lead to severe pain, frequent infections and permanent scarring.
Furthermore, Vyjuvek doesn’t require supportive equipment or technology or specialized expertise per se, which may eventually make it accessible even to patients who live far from treatment centers. Nevertheless, DEB is a very rare disease. Approximately 3,000 patients suffer from it in the U.S. As such, despite the high cost per patient, it’s unlikely to expose individual payers to substantial outlays.
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