资讯

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 ...
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 ...
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] ...
GlobalData on MSN2 天
Ultragenyx receives complete response letter for UX111 BLA
The FDA's request stems from observations made during recent inspections of Ultragenyx's manufacturing facilities.
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.
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 ...
Investor's Business Daily on MSN2 天
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.
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 ...
BioSpace19 小时
FDA Layoffs, 200 Rejection Letters, User Fees and Priority Vouchers, Bad Week for Rare Disease
Around 3,500 FDA employees received termination emails; FDA Commissioner Marty Makary suggests lowering industry user fees ...
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 Pharmaceutical, Apple And Other Big Stocks Moving Lower In Monday's Pre-Market ...
Ultragenyx received a Complete Response Letter from the FDA for UX111 AAV gene therapy to treat Sanfilippo Syndrome Type A ...
BioWorld2 天
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 ...