Olivia had been showing symptoms since she was just 7 months old — including a belly that remained swollen, hard to the touch ...

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

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

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

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

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

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