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health23h ago
They're in a race to save their children's lives. A newly approved drug has given them hope.
- The FDA granted accelerated approval for Avlayah, a new enzyme replacement therapy for Hunter syndrome, aiming to slow cognitive decline.
- Avlayah is the first FDA-approved Hunter syndrome treatment in two decades that penetrates the blood-brain barrier.
- Early treatment may extend life and prevent cognitive decline if started before brain damage occurs.
- The drug targets cognitive decline, addressing a key unmet need in Hunter syndrome.
- Families like Kim Stephens and Roran Jaskulski hope for stability and improved quality of life for their children.
- The current standard treatment helps physical symptoms but does not halt cognitive decline.
- Denali says it is engaging with payers to ensure rapid access to Avlayah for families.
- The drug’s approval reveals ongoing FDA debates over rare disease treatments amid patient advocacy pressure.
- The therapy marks progress in addressing neurodegenerative aspects, a breakthrough for families and researchers.
- The drug Avlayah is priced at a list price of $5,200 per 150-milligram vial, with Denali prioritizing access.
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