Ultragenyx unveils restructuring plan, laying off 10% of workforce

Ultragenyx has initiated a strategic restructuring plan, reducing headcount and expenses, to focus resources on what the company sees as its “largest value drivers.”

As part of the company’s financial update, Ultragenyx announced a 10% workforce reduction, impacting approximately 130 employees. The company’s net loss for the fourth quarter 2025 was $129 million; net loss for the year was $575 million.

“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, MD, Ph.D., CEO and president of Ultragenyx.

Ultragenyx completed a rolling BLA submission for its AAV8 gene therapy for glycogen storage disease type Ia, DTX401, in December 2025. The therapy has a PDUFA date in the third quarter of 2026.

Ultragenyx also revealed that it had recently received an incomplete response letter (IRL) regarding its resubmitted its BLA seeking accelerated approval for its AAV9 gene therapy, UX111, as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA). The IRL requests additional supportive documentation related to its CRL CMC responses, which the company says it will provide in a resubmission.

Ultragenyx had resubmitted the BLA for UX111 earlier this month, after getting hit with a CRL in July 2025. The resubmission contained substantial longer-term data on multiple measures of neurologic benefit to support an intermediate clinical endpoint for accelerated approval supported further by CSF heparan sulfate and other biomarker data, as agreed with the FDA during the last clinical review. It also included comprehensive responses to CMC-related observations outlined in the CRL.