The FDA requires the initiation of Phase III trials, uniQure's gene therapy for Huntington's disease halted market listing, stock price plummeted by approximately 33%

Gene therapy company uniQure (QURE.US) had originally hoped that its Huntington's disease candidate drug AMT-130 would receive accelerated approval from the US FDA, but this hope was recently dashed. The FDA clearly informed this biopharmaceutical company that a randomized, double-blind, placebo-controlled Phase III clinical trial is needed. In response to this news, the company's stock price dropped by approximately 33% at the close of trading on Monday. According to a press release from the company, uniQure had planned to seek approval based on data from a Phase I/II study comparing to an external control. However, the FDA believes that the existing data is not sufficient to serve as the "primary evidence of effectiveness required to support a marketing application for AMT-130". uniQure stated that they will request a Type B meeting with the FDA in the second quarter to discuss the next steps. During a subsequent conference call, CEO Matthew Kapusta revealed that the Phase I/II data showed that the drug could slow disease progression by 75% and slow the decline in overall function by 60%. However, he also acknowledged that during a pre-BLA meeting held in October of last year, the FDA had already indicated that the data submitted at that time was unlikely to serve as the primary basis for a BLA application.