FDA approves Ultragenyx gene therapy for rare childhood disease

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Go back to FDA approves Ultragenyx gene therapy for rare childhood disease

Ultragenyx Announces Approval of FAYUVI™ Gene Therapy, the First-Ever FDA-Approved Treatment for Sanfilippo Syndrome Type A (MPS IIIA)

September 17, 2026 3:48 PM EDT

FAYUVI is a highly anticipated, first-ever treatment option with the potential to stop or slow the devastating, irreversible neurologic progression and loss of function associated with Sanfilippo syndrome Type A

Ultragenyxs UltraCare® program will support access, and commercial product is expected to be available to ship to Qualified Treatment Centers within 30-60 days

FAYUVI marks the second gene therapy approval, and sixth FDA approval overall, for Ultragenyx

The Company received a Priority Review Voucher upon FAYUVI approval

Ultragenyx to Host Conference Call... More