Gene therapy firm uniQure NV (QURE.US) saw its hopes for accelerated approval of its Huntington's disease candidate AMT-130 by the U.S. FDA dashed recently. The FDA has clearly informed the biopharmaceutical company that it must conduct a randomized, double-blind, sham-surgery controlled Phase III clinical trial. Following this news, the company's stock closed down approximately 33% on Monday. According to a press release from the company, uniQure had previously planned to seek approval based on data from a Phase I/II study compared with an external control. However, the FDA determined that the existing data is still insufficient to "serve as the primary evidence of effectiveness required to support a marketing application for AMT-130." uniQure stated it will request a Type B meeting with the FDA in the second quarter to discuss the subsequent development path. In a follow-up conference call, CEO Matthew Kapusta revealed that Phase I/II data showed the drug could slow disease progression by 75% and reduce the decline in total functional capacity by 60%. However, he also acknowledged that during a pre-BLA meeting last October, "the FDA had already indicated that the data submitted at that time... was unlikely to serve as the primary basis for supporting a BLA submission."