All articles tagged with #huntingtons disease
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.
FDA vaccine chief Dr. Vinay Prasad is leaving the agency at the end of April to return to UCSF, marking a second departure in under a year amid ongoing controversies over vaccine and rare-disease drug reviews, including disputes with Moderna over an mRNA flu vaccine and with UniQure on a Huntington's disease gene therapy.
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.
New population-genomics research shows that many so-called monogenic diseases do not have 100% penetrance; a person carrying a disease-associated variant may remain healthy depending on other genetic factors and environment. Large datasets reveal that variants once believed to almost always cause conditions like inherited retinal degenerations, thyroid cancer, ovarian insufficiency, and even Huntington's disease do not guarantee disease in the general population, highlighting the need for nuanced genetic counseling and individualized risk assessments and potentially informing advances in gene therapies and embryo selection.
A small trial of uniQure’s AMT-130 gene therapy in 29 Huntington’s disease patients showed reduced production of the mutant huntingtin protein, slower cognitive decline, and lower levels of neurofilament light in cerebrospinal fluid over three years, suggesting a potential disease-modifying effect. Results were reported in a press release and have not yet undergone peer review, but they point to a possible treatment that could be used earlier in the disease course, while researchers continue to identify early biomarkers and expand the treatment window before motor symptoms appear.
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 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.
The Harvard Brain Tissue Resource Center faces potential setbacks in Huntington's disease research due to funding cuts that threaten the timely collection and preservation of donated brain tissue, which is crucial for ongoing studies.
A small, early study suggests that the gene therapy AMT-130 may slow Huntington's disease progression, but results are preliminary, unreviewed, and require further research to confirm efficacy and safety.
This week in science, a groundbreaking gene therapy for Huntington's disease was announced, a record-breaking tropical storm hit Asia, a black hole exceeded theoretical growth limits challenging current cosmological models, and research linked air pollution to increased psoriasis cases worldwide.
A small trial has shown that a one-time gene therapy can significantly slow the progression of Huntington’s disease by 75% over three years, offering new hope for treatment, though further research is needed to confirm these preliminary results.
Researchers in the UK have trialed a promising gene therapy, AMT-130, which can slow Huntington's disease progression by up to 75% over three years, potentially offering the first effective treatment for this fatal neurodegenerative disorder.