Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) on Thursday reported mixed fourth-quarter earnings and initiated a strategic restructuring plan.
“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, CEO and president 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,” Kakkis commented on Thursday.
FDA Update
Tucked in its earnings press release, the company said it received an Incomplete Response Letter (IRL) from the U.S Food and Drug Administration (FDA) regarding its resubmitted marketing application.
The application seeks approval for UX111 (rebisufligene etisparvovec) AAV9 gene therapy for Sanfilippo syndrome type A (MPS IIIA).
The IRL requests additional supportive documentation related to its CRL CMC responses, which the company will provide in a resubmission.
William Blair wrote, “While the IRL is frustrating, …