Ultragenyx Q2 Revenue Jumps 28% to $214M; Two Gene Therapy Approvals Loom with PDUFA Dates in August and September
RARE sits 37% above its 52-week low of $18.29.
Summary
Ultragenyx posted Q2 2026 revenue of $214M, up 28% year-over-year, and a narrower net loss of $92M. Two gene therapy candidates have near-term PDUFA dates: DTX401 (Aug 23) and UX111 (Sep 19). Cash stands at $436M.
Key Events · Earnings and Guidance · RARE
-
Record Quarterly Revenue
Total revenues reached $214M in Q2 2026, a 28% increase from $167M in Q2 2025, driven by Crysvita product sales ($54M, +54%) and royalty revenue ($102M, +19%).
-
Narrower Net Loss
Net loss improved to $92M from $115M a year ago, reflecting revenue growth and cost discipline from the February 2026 restructuring.
-
Cash Position and Burn
Cash, equivalents, and marketable securities totaled $436M at June 30, 2026, down from $737M at year-end 2025. Operating cash burn was $294M in H1 2026.
-
Two Near-Term PDUFA Catalysts
DTX401 (GSDIa) has an August 23, 2026 PDUFA date; UX111 (MPS IIIA) has a September 19, 2026 PDUFA date. Both are under Priority Review and could become the company's first gene therapy approvals.
Analysis · RARE · Life Sciences
Record quarterly revenue of $214 million, fueled by a 54% surge in Crysvita product sales and a 19% rise in royalty revenue, drove Ultragenyx's top line. The net loss narrowed to $92 million from $115 million a year ago, aided by that growth and a cost-cutting restructuring. More critically, the company stands on the verge of two potential gene therapy approvals: DTX401 for GSDIa carries an August 23 PDUFA date, while UX111 for Sanfilippo syndrome type A has a September 19 date, both under Priority Review. Cash reserves of $436 million provide runway, though the burn rate remains steep at $294 million in operating cash used in the first half. These upcoming catalysts could reshape the revenue base and validate the gene therapy platform.
At the time of this filing, RARE was trading at $25.13 on NASDAQ in the Life Sciences sector, with a market capitalization of approximately $2.5B. The 52-week trading range was $18.29 to $39.89. This filing was assessed with positive market sentiment and an importance score of 8 out of 10.