Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) just added another commercial product to its portfolio. 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. Fayuvi becomes the first approved treatment for this rare and fatal childhood disorder.
Ultragenyx stock jumped more than 12% following the announcement of the Fayuvi approval. But the stock is still down nearly 40% this year. The more important question for investors right now is what the approval changes about Ultragenyx's earnings trajectory.
The answer is that Fayuvi expands Ultragenyx's commercial portfolio just as the company is working toward profitability in 2027. Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) made a loss of $575 million on revenue of $673 million in 2025. The loss widened slightly from $569 million in 2024, and the revenue increased from $560 million.
This year, the company guided for $730 million to $760 million in total revenue from its current products, excluding potential revenue from new product launches such as Fayuvi. With Ultragenyx aiming to reach profitability in 2027, the Fayuvi approval may have just strengthened that profitability target. That makes the drug's approval more than a regulatory milestone for the company.
Ultragenyx expects Fayuvi to be available to patients within 30 to 60 days. That short availability timeline means the company will begin testing actual commercial demand relatively quickly. The pricing offers a glimpse into Fayuvi's potentially material contribution to earnings.
Ultragenyx has set a list price of $3.95 million for a one-time treatment with Fayuvi in the US. That makes Fayuvi one of the world's most expensive drugs. JP Morgan estimates that Fayuvi could bring in $200 million to $250 million in peak global sales.
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