The company now says that, during a recent meeting with the FDA, the two parties agreed on some key elements for an accelerated approval application.

In a Type B meeting, the FDA signified that it will allow uniQure to use a natural history control, the composite Unified Huntington’s Disease Rating Scale, and neurofilament light chain levels to support the accelerated approval of its gene therapy AMT-130.

Crucially for the Netherlands-based biopharma company, the FDA has said that the composite Unified Huntington's Disease Rating Scale (cUHDRS) can serve as an intermediate clinical endpoint for its review, along with the use of neurofilament light chain (NfL) levels in cerebrospinal fluid (CSF) as a biomarker for efficacy.