FDA Approves Ultragenyx's Fayuvi, First Treatment for Sanfilippo Syndrome Type A

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The FDA on September 17 approved Fayuvi for the treatment of neurologic manifestations of Sanfilippo syndrome Type A in pediatric patients with preserved neurodevelopmental function, making it the first approved treatment for the rare and fatal childhood disorder. Ultragenyx Pharmaceutical Inc. expects Fayuvi to be available to patients within 30 to 60 days, and has set a list price of $3.95 million for a one-time treatment in the US, one of the world's most expensive drugs. JP Morgan estimates Fayuvi could bring in $200 million to $250 million in peak global sales, which at the lower end would represent around 26% of Ultragenyx's 2026 revenue guidance midpoint. The approval expands Ultragenyx's commercial portfolio as the company works toward profitability in 2027, after it posted a loss of $575 million on revenue of $673 million in 2025 and guided for $730 million to $760 million in total revenue this year from current products, excluding potential revenue from new launches such as Fayuvi. Ultragenyx stock jumped more than 12% on the approval but remains down nearly 40% this year, while short interest stood at 17.1 million shares, or 18% of the public float, as of August 31.

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FDA approved Fayuvi, the first treatment for Sanfilippo syndrome Type A, expanding Ultragenyx's commercial portfolio.

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