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

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

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

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

RARE faces regulatory setback as FDA issues a CRL for UX111, delaying approval of the MPS IIIA gene therapy until 2026.

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

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

Ultragenyx Pharmaceutical has received a complete response letter (CRL) from the FDA concerning its BLA for UX111 (ABO-102).

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

Novato, California Monday, July 14, 2025, 16:00 Hrs [IST] ...

Ultragenyx Pharmaceuticals took another hit when the FDA rejected its gene therapy for patients with a rare, genetic disorder.

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