Ultragenyx Wins FDA Approval for FAYUVI, First Sanfilippo Type A Treatment

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Ultragenyx Pharmaceutical received full U.S. FDA approval in September 2026 for FAYUVI, also known as UX111, the first-ever treatment for pediatric patients with Sanfilippo syndrome Type A, a previously untreated ultra-rare and fatal neurodegenerative disease. The approval came with a Priority Review Voucher and expands Ultragenyx's commercial gene therapy portfolio and U.S. manufacturing footprint. The FAYUVI clearance follows the accelerated FDA approval of GENGLYCOS, also known as DTX401, for GSDIa in August 2026, giving the company two new gene therapies approved within weeks, each generating its own Priority Review Voucher. Ultragenyx's investment narrative projects $1.2 billion in revenue and $43.8 million in earnings by 2029, with a $27.00 fair value estimate implying 86% upside to the current price. The company's biggest immediate risk remains high cash burn and potential dilution if launches underperform or costs do not come down, with adoption, reimbursement and scaling of the new therapies shaping whether it can move closer to its 2027 profitability goal.

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FDA grants full approval for FAYUVI (UX111), the first Sanfilippo Type A treatment, expanding its commercial gene therapy portfolio

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