Amicus to pursue more migalastat data as FDA rules out quick path
Amicus Therapeutics (FOLD) plunged in after-hours trading after ruling out accelerated approval for its migalastat product and confirming the need for additional data. AMICUS SAYS MORE DATA NEEDED: In a press release shortly after market close, Amicus detailed its "planned regulatory pathway to collect additional data to support full approval" of migalastat: "Following several collaborative discussions with the FDA, including the receipt of final written minutes from an in-person Type B meeting, the company plans to collect additional data on gastrointestinal symptoms in Fabry patients who have an amenable mutation. Key elements of the additional data generation... may include: Randomized, 12-month, placebo-controlled pivotal 'cross-over' study in treatment naive Fabry patients who have an amenable mutation and GI symptoms. Crossover study design provides sufficient powering with a small number of patients, ~35 patients planned. Primary endpoint to assess diarrhea based upon established FDA irritable bowel syndrome guidance." Amicus noted it is working with the FDA to finalize the clinical protocol and plans to initiate enrollment in 2017, with data expected in 2019. The company also disclosed that "during its review of the briefing document submitted and in discussions with Amicus, the FDA acknowledged that significant unmet medical need exists in Fabry disease. The agency also indicated that kidney globotriaosylceramide is currently not considered a basis for an accelerated approval under Subpart H." Weighing in on the news, CEO John Crowley stated, "While we believe that the totality of the data from our studies with migalastat support the submission of a new drug application today, we acknowledge the FDA's position that accelerated approval based on kidney GL-3 reduction is not currently an option... FDA has been flexible in allowing a crossover design and in our use of established GI endpoints to measure clinical benefit in Fabry patients. We are fully committed to the additional work necessary to move migalastat toward approval." PRICE ACTION: Shares of Amicus are down 27.76% to $6.01 in post-market trading.