FDA not ready to give accelerated approval for Pompe disease drug

US biotech Amicus Therapeutics (Nasdaq: FOLD) suffered a second setback in its bid for speedy approval for its Pompe diseases candidate, with the US Food and Drug Administration being negative following similar action from European regulators in June, seeing the firm’s shares close down 4.0% at $12.00 on Monday.

During the third quarter, Amicus held a Type C meeting with the FDA in order to discuss the regulatory path for AT-GAA for Pompe disease. Specifically, Amicus sought input on the design of a pivotal study for full approval for AT-GAA, other supplemental clinical studies in Pompe disease patients, and whether Amicus may pursue an Accelerated Approval pathway in the USA at this time.

Amicus has now received final written minutes from the Type C meeting, in which the FDA noted “the importance of expediting new treatments to Pompe patients as fast as possible.” Amicus has incorporated key elements of feedback from the FDA, including the Type C meeting, along with the prior scientific advice received from the European Medicines Agency and plans to initiate a pivotal study in second-half 2018.