STAT+: FDA approves Denali Therapeutics drug for Hunter syndrome
Why it matters: A new Hunter syndrome drug offers hope, despite the FDA's tougher stance on rare disease therapies.
- FDA approved Denali Therapeutics' drug, Avlayah, for Hunter syndrome, a notable decision given recent rejections of rare disease therapies.
- Regenxbio's Hunter syndrome gene therapy was rejected by the FDA last month due to a need for more clinical data, setting a higher bar for evidence.
- Sarepta Therapeutics shares rose on early promise for rare disease drugs, indicating broader market interest and potential for other treatments in this space, as reported by STAT News.
The FDA has approved Denali Therapeutics' drug, Avlayah, for Hunter syndrome, a significant decision given the agency's recent stricter stance on rare disease therapies, including the rejection of Regenxbio's gene therapy for the same condition. This approval offers a new treatment option for Hunter syndrome patients, contrasting with the higher data standards now expected for other rare disease drugs.
