FDA Approves Ultragenyx's Fayuvi for Sanfilippo Syndrome — SkimNews
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- FDA approved Ultragenyx's gene therapy Fayuvi on Thursday for Sanfilippo syndrome type A, an ultra-rare disease sometimes called childhood Alzheimer's.
- Fayuvi is the first drug specifically approved to treat Sanfilippo, developed by Ultragenyx.
- Ultragenyx did not immediately release pricing for the therapy following the approval.
- Cara O'Neill, chief science officer of the Cure Sanfilippo Foundation, said the approval means families would finally receive "hope and an action plan for treatment" rather than being told to "take their kids home and love them."
Why it matters: For a disease with no prior FDA-approved therapy, this shifts the standard of care from palliative guidance to active treatment — but with Ultragenyx not yet disclosing pricing, families and payers face near-term uncertainty about actual access costs.
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