Shares of uniQure (QURE) skyrocketed 93.7% in pre-market trading on Tuesday, following the company's announcement that it has reached an agreement with the U.S. Food and Drug Administration on key elements of an accelerated approval pathway for its investigational gene therapy AMT-130 for Huntington's disease.

In a major regulatory milestone, the FDA has agreed that data from uniQure's ongoing Phase 1/2 clinical studies of AMT-130, compared to an external natural history control group, may serve as the primary basis for a Biologics License Application submission under the accelerated approval pathway. This means uniQure can potentially avoid the need for an additional pre-submission study before filing for approval.

The FDA also agreed that the composite Unified Huntington's Disease Rating Scale (cUHDRS) may be used as an intermediate clinical endpoint for accelerated approval, and that reductions in neurofilament light chain measured in cerebrospinal fluid could serve as supportive evidence of therapeutic benefit. AMT-130 was previously granted Regenerative Medicine Advanced Therapy designation by the FDA in May 2024, recognizing its potential to address an unmet medical need for Huntington's disease.