Ultragenyx Pharmaceuticals RARE announced that the FDA has issued a Complete Response Letter (CRL) for its biologics license ...

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

While the CRL derails a near-term clearance, one analyst described the rejection as more of “a speed bump to approval, rather ...

Ultragenyx Pharmaceuticals took another hit when the FDA rejected its gene therapy for patients with a rare, genetic disorder.

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

Ultragenyx received a Complete Response Letter from the FDA for UX111 AAV gene therapy to treat Sanfilippo Syndrome Type A ...

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

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 ...

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the stocks drowned heavily. Are you holding any? Ultragenyx fell by 26 ...

Olivia had been showing symptoms since she was just 7 months old — including a belly that remained swollen, hard to the touch and rounder than it should have been. Her pediatrician brushed it off as a ...

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

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