Huntington's disease is a hereditary, currently incurable neurological disorder affecting approximately 75,000 people in the United States, Europe, and the United Kingdom, according to uniQure. Effective therapies against the disease progression do not yet exist, according to the company. On September 2, 2026, the company announced that it had submitted a regulatory application to the FDA.
Extended Three-Year Data Show Significant Slowing
According to an extended three-year analysis released by uniQure, disease progression slowed by 80 percent in 15 high-dose patients compared to matched controls without treatment, based on the cUHDRS composite score, and by 67 percent based on daily functioning as measured by the TFC scale. Both values reached statistical significance, according to a report by PET on October 12, 2026.
Four Years After Treatment: A Weaker Signal
At the end of September, the industry news service STAT reported that a new analysis showed a 44 percent slowing of Huntington's progression at four years in the high-dose group compared to an external control group from a natural history study. The difference was not statistically significant. The trade publication HDBuzz urged caution in interpreting this value because of the small group of 12 people followed over four years. The high-dose group performed better than the equally small low-dose group, suggesting a dose-dependent effect. For daily functioning, the TFC scale showed a significant 61 percent slowing of decline in the high-dose group compared to controls. For movement symptoms and a thinking test, however, AMT-130 did not appear to slow changes compared to controls, while a reading and thinking test showed significant slowing. The biomarker neurofilament-light increased only 4 percent over four years in high-dose patients, whereas it normally increases 10 to 15 percent annually in progressive Huntington's disease.
Why Control Groups Differ
According to PET, after four years, more than half of the control participants' data were missing. Patients with faster deterioration more frequently discontinued follow-up.
The ongoing regulatory application continues to rely exclusively on the three-year data from the initial 12 patients, which the FDA agreed could be used as the primary basis for an expedited approval application, according to company statements made public in June 2026. The company plans to present the new results at a future meeting with the agency, according to PET.