Fayuvi Adds a New Revenue Stream, but Ultragenyx’s 2027 Goal Faces Pressure

The FDA approved Ultragenyx’s Fayuvi gene therapy on September 17 for children with Sanfilippo syndrome type A, the first approved treatment for the disease. Shares rose 12.6% that day to $14.50.

Fayuvi is a one-time infusion for pediatric patients whose neurodevelopment is preserved. Ultragenyx set its U.S. list price at $3.95 million per treatment and expects availability at specialist centers within 30 to 60 days, according to Reuters. J.P. Morgan analysts estimate peak global sales of $200 million to $250 million.

That potential revenue could help, but it will not immediately erase the company’s losses. Ultragenyx reported a $92 million net loss in the second quarter, with $289 million in operating expenses and $97 million in cash used for operations.

Before Fayuvi’s approval, the company forecast 2026 revenue of $730 million to $760 million, excluding possible new launches. It also expected research and selling expenses in 2027 to fall at least 15% from 2025 levels.

But that profitability outlook faces a fresh complication. On September 2, Ultragenyx said its Phase 3 Aspire trial of GTX-102 for Angelman syndrome missed both its main and key secondary goals. The company said it would assess the program and make significant expense reductions.

The latest explicit reaffirmation of the 2027 target came in the August 4 earnings update, before the trial failure. Ultragenyx has not disclosed how the GTX-102 decision will affect projected savings, leaving investors without a revised profitability outlook.

Fayuvi’s launch now offers a new source of potential growth, but uptake, insurance coverage and treatment-center readiness will determine how quickly sales arrive. The FDA’s label also warns of serious risks, including thrombotic microangiopathy, and requires close monitoring after infusion.