Competition from Amgen's romosozumab and patient preference for subcutaneous administration threaten setrusumab’s potential ...

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 decision follows the initial approval granted in September 2023, which covered patients aged five years and above.

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

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 ...

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.

Del-zota is designed for patients with mutations amenable to exon 44 skipping, which is just 7% of the overall DMD patient ...

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

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

Nebius Inc. (NASDAQ: NBIS) soared 17% on Monday after Goldman Sachs initiated coverage with a “Buy” rating and a $68 price target — implying roughly 39% upside from current levels. The investment bank ...