uniQure Aligns with FDA on Accelerated Approval Pathway, Raising Analyst Expectations

Netherlands-based uniQure NV (NASDAQ: QURE), a pioneer in gene therapy, has achieved yet another milestone in the development of its experimental Huntington’s disease treatment, AMT-130. On December 14, 2024, the company announced that it had aligned with the U.S. Food and Drug Administration (FDA) on key aspects of an Accelerated Approval pathway, potentially fast-tracking the therapy’s market entry. This announcement follows the RMAT (Regenerative Medicine Advanced Therapy) designation granted to AMT-130 in May 2024 and promising interim data in July showing a durable, dose-dependent slowing of disease progression. AMT-130, one of uniQure’s flagship assets, is designed to enter brain cells and block the genetic instructions for producing the harmful protein that drives Huntington’s disease.

The Accelerated Approval pathway once granted could be a game-changer for uniQure, positioning the company at the forefront of the race to develop effective treatments for this condition. Analysts have revised their projections sharply upward since the December announcement, with Visible Alpha consensus now forecasting peak global sales of $742 million by 2033, compared to $417 million previously. The drug’s probability of success has also risen to 43%, from 31%.

Huntington’s disease, a genetic disorder that progressively damages nerve cells in the brain, leads to severe impairments in movement, cognition, and mental health. While symptomatic treatments exist, there are no approved therapies to slow disease progression—a void AMT-130 aims to fill.