Manufacturing issues are the latest problem for Ultragenyx Pharmaceutical Inc. to solve after last week’s disappointment in a phase III study to treat brittle bones. The U.S. FDA gave the company a ...

The firm said the agency cited chemistry, manufacturing, and controls issues in its complete response letter, but had no concerns about product quality.

The FDA cited manufacturing issues but did not flag problems with Ultragenyx’s data package for UX111, with the biotech ...

The FDA's request stems from observations made during recent inspections of Ultragenyx's manufacturing facilities.

Novato, California Monday, July 14, 2025, 16:00 Hrs [IST] ...

Ultragenyx Pharmaceutical (Nasdaq: RARE) has been dealt a regulatory setback, as the American medicines regulator has ...

The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's experimental gene therapy to treat ...

Investing.com -- Ultragenyx Pharmaceutical (TADAWUL: 2070) Inc. (NASDAQ: RARE) stock fell over 5% after the company announced that the U.S. Food and Drug Administration (FDA) has issued a Complete ...

Complete Response Letter (CRL) cited specific chemistry, manufacturing and controls (CMC) related observations that are ...

In March 2022, Olivia Stoop was diagnosed with Sanfilippo syndrome, a terminal genetic disorder Leading up to her daughter Olivia’s 2-year checkup, Erin Stoop couldn’t shake the feeling that ...