Fierce Biotech: Ultragenyx wins surrogate endpoint debate, securing green light to seek FDA approval of gene therapy
Ultragenyx Pharmaceutical's work to get the FDA to recognize a biomarker as a surrogate endpoint looks to have paid off. With the agency agreeing the biomarker is a reasonable surrogate endpoint, Ultragenyx is preparing to file for approval of its Sanfilippo syndrome gene therapy around the end of the year.