News

US FDA declines to approve Ultragenyx's gene therapy for rare genetic disorder
The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's experimental gene therapy to treat ...
FDA Delays Approval For Ultragenyx's Gene Therapy, But Not A Dealbreaker, Says Analyst
Ultragenyx's UX111 gene therapy faces FDA delay due to facility issues; analysts adjust price targets amid uncertain ...
GlobalData on MSN21h
Ultragenyx receives complete response letter for UX111 BLA
The FDA's request stems from observations made during recent inspections of Ultragenyx's manufacturing facilities.
Fierce Pharma18h
Ultragenyx takes another hit as FDA snubs AAV gene therapy over manufacturing issues
The manufacturing-related rejection for UX111 is more than a setback for Ultragenyx, as it also delays a key test of the new ...
FDA rejects Ultragenyx gene therapy over manufacturing concerns
While the complete response letter derails a near-term clearance, one analyst described the rejection as more of “a speed ...
US FDA declines to approve Ultragenyx's gene therapy for rare inherited disorder
The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's experimental gene therapy to treat ...
BioSpace20h
Ultragenyx Takes Second Hit as FDA Rejects Gene Therapy for Sanfilippo Syndrome
The FDA cited manufacturing issues but did not flag problems with Ultragenyx’s data package for UX111, with the biotech ...
Ultragenyx Pharmaceutical, Apple And Other Big Stocks Moving Lower In Monday's Pre-Market Session
Ultragenyx received a Complete Response Letter from the FDA for UX111 AAV gene therapy to treat Sanfilippo Syndrome Type A ...
STAT18h
FDA declines to approve Ultragenyx gene therapy
In his first 100 days as commissioner of the Food and Drug Administration, Marty Makary has made a handful of splashy ...
Ultragenyx: 'Hold' The Best Strategy As Stock Tanks On Bone Disease Study Miss
Ultragenyx Pharmaceutical Inc. shares plunged 30% as UX143 trial raises efficacy doubts. Click for more on RARE and my ...
The Pharma Letter23h
Ultragenyx hit with FDA delay for gene therapy targeting rare childhood disorder
Ultragenyx Pharmaceutical (Nasdaq: RARE) has been dealt a regulatory setback, as the American medicines regulator has ...
BioWorld10h
Ultragenyx hit again, this time with a CRL in rare disease
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 ...