Amicus Therapeutics, Inc. (FOLD) is watching its shares drop on Tuesday after the company provided an update on its regulatory pathway for its Fabry Disease treatment. After the company had several collaborative discussions with the U.S. Food and Drug Administration (FDA), Amicus plans to collect additional data on gastrointestinal (GI) symptoms in Fabry patients who have an amenable mutation.
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 (GL-3) is currently not considered a basis for an accelerated approval.
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More than 50% of patients with Fabry disease report or show GI signs and symptoms, including diarrhea, abdominal pain, constipation, nausea, and vomiting. Amicus previously presented positive GI data in a completed Phase 3 randomized, placebo controlled Study 011 (FACETS) in treatment-naïve Fabry patients with amenable mutations.
The GI data from this study showed a significant decrease in diarrhea in patients with amenable mutations treated with migalastat versus placebo, which persisted after 18-to-24 months of treatment with migalastat.
John F. Crowley, Chairman and CEO of Amicus, commented:
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. We have thus defined a plan to collect additional GI data to support full approval for migalastat that we believe is feasible in a reasonable amount of time and with a high likelihood of success based on positive GI data generated in our previous Phase 3 Study 011. 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 in the United States.
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Shares of Amicus were last trading down about 22% at $6.49, with a consensus analyst price target of $12.38 and a 52-week trading range of $4.98 to $11.02.