A gene therapy for Huntington’s disease has a new path to Food and Drug Administration approval after the ouster of several Trump officials, particularly Vinay Prasadwho rejected the therapy in a shocking move that one former FDA official called “truly evil.”
Huntington’s disease is an inherited condition that usually appears in middle age and causes nerve cells in the brain to gradually break down. Currently there are no treatments for the disease and many of those affected die between the ages of 50 and 60.
Gene therapy company UniQure has developed a unique treatment, AMT-130, that aims to reduce brain levels of the mutant protein behind the disease, called Huntingtin. Data from a small initial trial suggested the drug could Delay disease progression by up to 75 percent.and patients and their advocates have closely followed the drug’s development with hopeful anticipation.
In 2024, the FDA told UniQure that it could apply for accelerated approval of AMT-130 without a placebo control group in its trial. While having a placebo control provides a high-quality comparator in a trial, it raised unique ethical concerns for UniQure’s gene therapy. Administration of AMT-30 requires 10 to 12 hours of brain surgery, meaning that a placebo control arm of a trial would require patients in a control group to undergo lengthy sham surgery that could involve drilling a shallow hole in their skulls.
UniQure moved forward without a placebo control, using untreated outpatients as a comparison control group for its trial, believing the FDA supported the plan. But during Prasad’s tenure as the FDA’s top gene therapy regulator, the agency scrapped the deal and demanded that UniQure perform mock surgeries as controls.
