Ultragenyx Cuts 10 Percent of Staff as Part of Restructuring

Rare Daily Staff

Ultragenyx said it has initiated a strategic restructuring plan that will eliminate 10 percent of its workforce, or about 130 people, cut expenses, and focus resources on its largest value drivers as it released its results for 2025.

The company said the reduction and partial reinvestment of expenses, along with planned growth in revenue from current and new product launches, are designed to keep the company on its path to profitability in 2027.

Ultragenyx provided updates on several key programs. It said the U.S. Food and Drug Administration is expected to act on its application to market its AAV8 gene therapy for glycogen storage disease type Ia (GSDIa) in the third quarter of 2026.

The company also resubmitted to the FDA its application to market its AAV9 gene therapy for Sanfilippo syndrome type A (MPS IIIA). The updated application included substantially longer-term data—recently presented at the 2026 WORLDSymposium—on multiple measures of neurologic benefit to support an intermediate clinical endpoint for accelerated approval. It is also supported by cerebrospinal fluid heparan sulfate and other biomarker data, as agreed with the FDA during the last clinical review. The FDA issued an incomplete response letter regarding the resubmission and asked for additional supportive documentation, which the company said it will provide in a revised filing.

Data from the fully enrolled, pivotal phase 3 ASPIRE study of its antisense oligonucleotide treatment for Angelman syndrome in patients with a genetically confirmed UBE3A deletion are expected in the second half of 2026. Enrollment in the phase 2/3 AURORA study is also underway in patients with other genotypes and ages, with the first patient dosed in October 2025.

Enrollment is complete for the fourth cohort in the ongoing dose-finding stage of the pivotal Cyprus2+ study of its AAV9 gene therapy for Wilson disease. Data from this stage are expected in 2026.

“The year ahead marks an important turning point for the company as we approach two potential product launches and a pivotal data readout that, together, could significantly accelerate our commercial revenue trajectory,” said Emil Kakkis, president and CEO of Ultragenyx. “We are implementing a strategic restructuring plan to reduce our operating expenses and ensure our resources are squarely aligned with our highest-impact opportunities, while leading the future of rare disease with multiple first-ever treatments.”