News

Ultragenyx Pharmaceuticals Gets CRL for UX111 Gene Therapy for MPS IIIA
RARE faces regulatory setback as FDA issues a CRL for UX111, delaying approval of the MPS IIIA gene therapy until 2026.
BioWorld2d
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 ...
Investor's Business Daily on MSN2d
Ultragenyx Pharma Takes Another Hit As FDA Slaps Its Gene Therapy With A Rejection
Ultragenyx Pharmaceuticals took another hit when the FDA rejected its gene therapy for patients with a rare, genetic disorder.
BioPharma Dive2d
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 ...
Precision Medicine Online2d
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.
BioSpace2d
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 receives complete response letter for UX111 BLA
The FDA's request stems from observations made during recent inspections of Ultragenyx's manufacturing facilities.
Pharmabiz2d
Ultragenyx receives CRL from US FDA for UX111 AAV gene therapy to treat Sanfilippo syndrome type A
Novato, California Monday, July 14, 2025, 16:00 Hrs [IST] ...
The Pharma Letter2d
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 genetic disorder
The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's experimental gene therapy to treat ...
Investing.com UK5d
Ultragenyx stock falls after FDA issues complete response letter for gene therapy
Investing.com -- Ultragenyx Pharmaceutical (TADAWUL: 2070) Inc. (NASDAQ: RARE) stock fell over 5% after the company announced that the U.S. Food and Drug Administration (FDA) has issued a Complete ...
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 ...