Days after FDA Commissioner Marty Makary appeared to malign uniQure’s AMT-130 in an interview with CNBC, the agency confirmed ...
Shares of uniQure plummeted more than 40% in premarket trading Monday after the company confirmed the FDA has rejected its current data package for AMT-130, a highly anticipated gene therapy for ...
In a strikingly blunt briefing, a senior FDA official accused uniQure of pushing “distorted” data to mask a “failed” therapy ...
While the FDA appears to be adamant that uniQure conduct a sham surgery–controlled Phase 3 trial before AMT-130 can be ...
UniQure needs to run another trial to prove its gene therapy "actually helps people with Huntington's disease," said a senior FDA official.
Successful program buildout prioritizes defined patient flow, infusion access, cold-chain capabilities, and accountability ...
Agency staff "strongly recommended" a sham surgery-controlled trial be conducted before an approval filing, a stance one analyst called a "worst case scenario" for UniQure.
Shares of uniQure BV (NASDAQ:QURE) surged 36% on Monday after reports emerged that the head of the U.S. Food and Drug Administration’s vaccines and gene therapies division will step down, triggering ...
March 11 (Reuters) - Regenxbio said on Wednesday that interim data from a early-to-mid stage study of its experimental gene therapy in patients with Duchenne muscular dystrophy showed continued ...