Ultragenyx Cuts Headcount After Gene Therapy Approvals

The Tip Desk · November 4, 2026 · 3 min read

Third-quarter revenue was $173 million as the company agreed to sell two Priority Review Vouchers for a combined $413 million and reaffirmed 2026 sales of $730 million to $760 million.

Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on rare genetic diseases, reported third-quarter 2026 revenue of $173 million and said it had received FDA approval of two gene therapies, GENGLYCOS and FAYUVI, in the quarter.

The result followed a second quarter that the company had called the highest quarterly revenue in its history. Crysvita, the largest franchise, generated $109 million in the third quarter, which the company said was consistent with expected ordering patterns.

Dojolvi revenue was $30 million. Evkeeza revenue was $24 million, which the company attributed to increased demand from new country launches and early access. Mepsevii revenue was $10 million.

The company reaffirmed full-year 2026 total revenue guidance of $730 million to $760 million, excluding revenue from new product launches. It also reaffirmed Crysvita guidance of $500 million to $520 million and Dojolvi guidance of $100 million to $110 million.

Net loss narrowed to $118 million, or $1.16 a share, from $180 million, or $1.81 a share, a year earlier. Research and development expense fell to $154 million from $216 million. Selling, general and administrative expense rose to $97 million from $87 million. Total operating expenses were $283 million, including $34 million of non-cash stock-based compensation.

In August, the FDA granted accelerated approval for GENGLYCOS, an AAV8 gene therapy for glycogen storage disease type Ia in patients eight years and older. In September, the FDA granted standard full approval for FAYUVI, an AAV9 gene therapy for pediatric Sanfilippo syndrome type A. Chief executive Emil D. Kakkis said the launches were progressing with growing demand.

In October, Ultragenyx entered two definitive agreements to sell the Priority Review Vouchers received with those approvals for a combined $413 million. The GENGLYCOS voucher sold for $210 million and closed; the FAYUVI voucher is priced at $203 million and is expected to close later in the month. Net proceeds from both sales are expected to be approximately $350 million.

With late-stage development programs completed, including those two first-ever FDA approvals, the company said it is entering a stage focused on commercial execution and a smaller R&D footprint. It initiated substantial headcount and expense reductions, including cutting the workforce by approximately 20% to about 1,000 employees, completing the wind-down of ongoing GTX-102 studies for Angelman syndrome, and terminating UX701 for Wilson disease after the Cyprus2+ dose-finding study did not meet the criteria to advance to a pivotal Phase 3.

Ultragenyx expects to reduce 2027 R&D expenses to less than $350 million and 2027 SG&A to less than $375 million, and said those actions are expected to drive at least a 50% reduction in 2027 R&D expenses versus 2025. Remaining R&D is focused on Phase 3 data for DTX301 in ornithine transcarbamylase deficiency, a potential path for UX143 in osteogenesis imperfecta, and measured spending on early-stage programs.

Fourth-quarter 2026 results are expected to include approximately $25 million of severance and other non-recurring restructuring charges. The reduction in force is expected to be substantially completed by the end of 2026.

Cash, cash equivalents, and marketable securities were $401 million as of September 30, 2026. Net cash used in operations was $43 million in the third quarter.

The company continues to anticipate achieving full-year GAAP profitability in 2027 and going forward.