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Ultragenyx wins FDA greenlight for first Sanfilippo therapy, priced at nearly $4M
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Fusion42 · 18 September 2026 · Fusion42 review
Ultragenyx has received FDA approval for Fayuvi, the first therapy for Sanfilippo syndrome type A, with a nearly $4 million price tag. This approval follows a previous FDA rejection in 2025 due to manufacturing issues and comes shortly after Ultragenyx's first gene therapy approval last month.
This Wire brief sits within Fusion42's coverage of Biotech, and 9 sources have reported it between 17 Sep 2026 and 28 Sep 2026.
◆ ◆ The Wire takeaway
You face a new benchmark for drug pricing in rare disease gene therapies as Ultragenyx prices its Sanfilippo treatment near $4 million. If you develop treatments in ultra-rare diseases, the pricing expectations and payer negotiations just moved sharply upwards.
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9 sources · first reported 17 Sep 2026 · latest 28 Sep 2026
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