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

British engineering giant Rolls-Royce announced on Tuesday a $75 million investment to expand its manufacturing operations in ...

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

This report also provides a detailed analysis of the current mucopolysaccharidosis marketed drugs and late-stage pipeline ...

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

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

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

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

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

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 ...