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FDA rejects Ultragenyx gene therapy over manufacturing concerns
While the CRL derails a near-term clearance, one analyst described the rejection as more of “a speed bump to approval, rather ...
Fierce Pharma2 天
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 ...
Zacks Investment Research on MSN1 天
Ultragenyx Pharmaceuticals Gets CRL for UX111 Gene Therapy for MPS IIIA
Ultragenyx Pharmaceuticals RARE announced that the FDA has issued a Complete Response Letter (CRL) for its biologics license ...
BioSpace2 天
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 ...
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 ...
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 ...
Ultragenyx Receives Complete Response Letter from FDA for UX111 AAV Gene Therapy to Treat ...
Complete Response Letter (CRL) cited specific chemistry, manufacturing and controls (CMC) related observations that are ...
The Pharma Letter2 天
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 ...
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 ...
STAT2 天
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 ...
Precision Medicine Online2 天
FDA Declines to Approve Ultragenyx's Gene Therapy for Sanfilippo Syndrome Type A
The firm said the agency cited chemistry, manufacturing, and controls issues in its complete response letter, but had no concerns about product quality.
Pharmabiz2 天
Ultragenyx receives CRL from US FDA for UX111 AAV gene therapy to treat Sanfilippo syndrome ...
Novato, California Monday, July 14, 2025, 16:00 Hrs [IST] ...