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

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) received a complete response letter from the FDA about UX111 for the treatment of Sanfilippo syndrome type A, citing concerns associated with chemistry, ...

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

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 ...

Around 3,500 FDA employees received termination emails; FDA Commissioner Marty Makary suggests lowering industry user fees ...

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 ...

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

Animal statues can be seen across downtown Pella, bringing a children’s story to life while also honoring special community ...

Ultragenyx's high burn rate, costly R&D, and recent setbacks increase dilution risk, impacting future profitability. Learn ...