All articles tagged with #uniqure
Vinay Prasad is set to leave the FDA for the second time, with his tenure marked by controversy, including a rejected mRNA vaccine, a controversial demand for an extra trial on a Huntington’s gene therapy from UniQure, public clashes with the company and alleged staff abuse, drawing mixed reactions from biotech investors and patient advocates as a replacement is named.
An FDA-organized media call featured a senior FDA official publicly attacking UniQure’s Huntington’s disease gene therapy, calling its data distorted and the therapy failed, a move seen as injecting politics into rare-disease regulation and potentially complicating oversight ahead of elections, with echoes of recent vaccine-review controversies and congressional pressure on the agency.
An FDA official held a rare press call to defend the agency’s decision to require an additional clinical trial for UniQure’s Huntington’s disease gene therapy, criticizing outside scientists and signaling concerns about potential conflicts of interest. The exchange follows ongoing uproar over the agency’s handling of drug approvals, including Moderna’s flu vaccine application, and underscores a tension between expanding rapid approvals and ensuring solid evidence through placebo-controlled trials in order to prevent unsafe or ineffective medicines.
An FDA official indicated UniQure’s Huntington’s disease therapy showed no demonstrated benefit based on current data, preventing a marketing application; the company disputes the assessment but hopes to align trial parameters for a potential future study.
The FDA rejected uniQure's plan to file AMT-130 using phase 1/2 data with an external control and demanded a prospective, randomized, sham-controlled phase 3 trial, dampening near-term approval hopes and triggering a strategic review by the company; uniQure will discuss trial designs with the FDA and add a four-year durability analysis to the phase 1/2 data.
Uniqure's stock dropped 59% after the FDA provided confusing and unexpected feedback regarding its Huntington's disease gene therapy, AMT-130, casting doubt on the company's plans for approval and indicating a key shift from previous communications.
UniQure's plans to submit its Huntington's disease gene therapy, AMT-130, for FDA approval are now uncertain after the agency indicated it no longer considers existing data sufficient, marking a significant shift from previous communications and causing a 60% drop in the company's premarket shares.
uniQure announced that the FDA no longer considers Phase I/II data versus an external control sufficient as primary evidence for a BLA submission for its Huntington's disease gene therapy AMT-130, creating uncertainty about the approval timeline. The company plans urgent discussions with the FDA and continues regulatory engagement in the EU and UK, despite holding breakthrough therapy and RMAT designations.
UniQure's experimental gene therapy, AMT-130, showed promising results in slowing Huntington's disease progression by 75%, leading to a over 240% surge in its stock price and plans to seek FDA approval by 2026, offering hope for a disease-modifying treatment.
Gene therapy company UniQure is undergoing a major revamp, including a 20% reduction in its workforce, the departure of its chief scientific officer, and a more than 50% cut in research and tech investment. The company aims to save $180 million over the next three years and extend its runway into the second quarter of 2027. A total of 114 positions will be eliminated.
Precision neuroscience startup Nido Biosciences has emerged from stealth mode with $109 million in funding and a Phase I study already underway. Belgian biotech Dualyx has raised $44 million in financing to take its first Treg candidate into a Phase I study in the second half of 2024. Gilead and Arcus are expanding their partnership to include inflammation targets. UniQure has sold part of its Hemgenix royalty rights for up to $400 million in cash. President Biden intends to nominate Monica Bertagnolli, currently leading the National Cancer Institute, as the head of the National Institutes of Health. The FDA has issued a complete response letter to Byondis, rejecting its pitch for a potential breast cancer therapy. Fulcrum Therapeutics has recruited Alex Sapir as CEO amid management upheaval and a full clinical hold for its sickle cell program. Verve Therapeutics has unveiled its second PCSK9-targeting program, Verve-102, as part of its wider third-quarter financial results.