FDA official calls UniQure's gene therapy a 'failed' treatment for Huntington's disease
#FDA #UniQure #gene therapy #Huntington's disease #failed treatment #regulatory approval #neurological disorder
📌 Key Takeaways
- FDA official labels UniQure's gene therapy for Huntington's disease as a 'failed' treatment
- The assessment highlights significant concerns over the therapy's efficacy
- This could impact regulatory approval and future development of similar gene therapies
- The statement underscores ongoing challenges in treating Huntington's disease
📖 Full Retelling
UniQure needs to run another trial to prove its gene therapy "actually helps people with Huntington's disease," said a senior FDA official.
🏷️ Themes
Regulatory Scrutiny, Gene Therapy, Neurological Disease
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Original Source
In this article UQ1-FF Follow your favorite stocks CREATE FREE ACCOUNT Thomas Fuller | SOPA Images | Lightrocket | Getty Images UniQure needs to run another study to prove that its gene therapy "actually helps people with Huntington's disease," a senior U.S. Food and Drug Administration official said on a call with reporters Thursday. The official, who requested anonymity before discussing sensitive information, confirmed the agency has asked the company to run a placebo controlled trial of its treatment, which is administered directly into the brain. UniQure has said that type of study isn't ethical because it would require putting people under general anesthesia for hours, a characterization the official disputed. "So what is really going on? UniQure is the latest company to make a failed therapy for Huntington's patients," the official said. "They likely acknowledge or understand at some deep level that their trial failed years ago, and instead of doing the right thing and running the correct clinical study, UniQure is performing a distorted or manipulated comparison in the mind of FDA." The comments mark the latest development in a messy public spat between UniQure and the FDA, and as the agency comes under fire for a number of recent drug approval application rejections, including some where companies have accused it of going back on previous guidance. FDA Commissioner Marty Makary in an interview with CNBC's Becky Quick last week seemingly criticized UniQure's gene therapy for Huntington's disease. Makary didn't name UniQure but described its treatment. watch now VIDEO 13:49 13:49 FDA Commissioner Dr. Makary on rare disease therapy approvals, internal politics at the agency Squawk Box UniQure then accused the FDA of reversing its stance that the company's clinical trial data would be sufficient to seek approval. UniQure's study used an outside database to measure how patients with Huntington's disease might decline without treatment, known as an external contr...
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