By Ben Glickman


Ultragenyx Pharmaceuticals said it will seek accelerated approval of its treatment for Sanfilippo syndrome after regulators approved the company's surrogate endpoint.

The Novato, Calif.-based pharmaceutical company said that the Food and Drug Administration had agreed that cerebral spinal fluid heparan sulfate would serve as a reasonable surrogate endpoint when evaluating UX111 AAV gene therapy. The company plans to file its biologics license application late this year or early next year.

Accelerated approval from the FDA allows companies to submit data on how their treatment affects a surrogate endpoint, which has to be pre-approved by regulators.

Ultragenyx said that it would need to finalize the details of its biologics license application with the FDA in a meeting before submitting.

Sanfilippo syndrome is a rare genetic disease affecting the central nervous system. It currently has no approved treatments, the company said, and is characterized by rapid neurodegeneration.


Write to Ben Glickman at ben.glickman@wsj.com

(END) Dow Jones Newswires

06-12-24 1626ET