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

Ultragenyx's UX111 gene therapy faces FDA delay due to facility issues; analysts adjust price targets amid uncertain ...

The manufacturing-related rejection for UX111 is more than a setback for Ultragenyx, as it also delays a key test of the new ...

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 ...

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

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

In his first 100 days as commissioner of the Food and Drug Administration, Marty Makary has made a handful of splashy ...

A minority of patients taking Ultragenyx Pharmaceutical’s Wilson disease gene therapy UX701 have come off standard-of-care drugs, leading the biotech to enroll a new cohort on a tweaked regimen ...

--Ultragenyx Pharmaceutical Inc., ... from the first three cohorts of the ongoing Phase 1/ 2 studies of DTX401, an investigational adeno-associated virus gene therapy ...

Ultragenyx said Thursday afternoon that its gene therapy for a rare liver ailment, glycogen disease type 1A, succeeded in a Phase 3 trial, setting up a potential approval. Patients with GSD1a, as ...

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