UniQure’s experimental gene therapy AMT-130 continued to slow Huntington’s disease progression four years after being surgically implanted into patients’ brains, the company reported. In the latest analysis, the high dose of the one-time treatment slowed progression by 44% compared with matched participants in an external control group based on a large natural history study. The difference was not statistically significant.
However, the magnitude of the treatment’s benefit has waned compared with a similar analysis conducted a year ago, which may raise questions about its durability just as the FDA begins reviewing UniQure’s marketing application. The new data therefore arrive at a pivotal moment for the program, with regulators now weighing whether the therapy should be approved despite the attenuated effect.