Drugs and FDA
UniQure gene therapy misses significance on four-year Huntington’s disease measure
At 48 months, AMT-130 was linked to 44% slower progression on cUHDRS, but the result was not statistically significant. TFC showed 61% slower progression.
UniQure reported 48-month results for AMT-130, its gene therapy for Huntington’s disease, from 12 people who received the high dose. Compared with an updated external patient database, disease progression on the cUHDRS measure was 44% slower, a result that did not reach statistical significance, the company said. The scale assesses cognition, motor skills and daily living.
On total functional capacity (TFC), a measure focused on daily activities, progression was 61% slower than in the external control, according to UniQure. The company said 53% of the reference data at 48 months was missing. In a post hoc analysis, UniQure reported slowing of 54% on cUHDRS and 68% on TFC.
UniQure has filed for accelerated FDA approval based on earlier 36-month data. The company’s chief medical officer said it was unclear whether the FDA would rerun the analysis using the new data.
