Amicus Therapeutics (FOLD) Reports Regulatory Pathway in U.S. for Migalastat for Fabry Disease

November 28, 2016 4:11 PM EST
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Amicus Therapeutics (Nasdaq: FOLD), a global biotechnology company at the forefront of rare and orphan diseases, announces its planned regulatory pathway to collect additional data to support full approval for the oral precision medicine migalastat for Fabry disease. Following several collaborative discussions with the U.S. Food and Drug Administration (FDA), including the receipt of final written minutes from an in-person Type B meeting, the Company plans to collect additional data on gastrointestinal (GI) symptoms in Fabry patients who have an amenable mutation.

Key elements of the additional data generation required for full approval of migalastat in the United States may include:

  • Randomized, 12-month, placebo-controlled pivotal “cross-over” study in treatment naïve 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 (IBS) guidance1
  • Amicus is working with FDA to finalize the clinical protocol and plans to initiate enrollment in 2017, with data expected in 2019
  • Upon successful NDA filing, the review will be based upon these new data and the totality of the data from all prior migalastat studies
  • Intermediate Expanded Access Program (EAP) planned to ensure short-term access to migalastat for U.S. patients who are currently on ERT and meet EAP requirements
  • U.S. patients currently enrolled in ongoing clinical extension studies will continue to receive migalastat

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 under Subpart H.

John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. 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. 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.”

More than 50% of patients with Fabry disease report or show GI signs and symptoms, including diarrhea, abdominal pain, constipation, nausea, and vomiting.2 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 (unadjusted p=0.03) in patients with amenable mutations treated with migalastat versus placebo during the 6-month double-blind phase (Stage 1), which persisted after 18-24 months of treatment with migalastat.

"There are significant unmet needs and a lack of treatment choices for people living with Fabry disease in the U.S.,” said Jack Johnson, Founder and Executive Director of the Fabry Support & Information Group (FSIG). “Amicus has been an outstanding partner for the Fabry community for more than a decade, incorporating the patient’s perspective throughout the entire migalastat development program and providing valuable research for the Fabry community. I look forward to the continued advancement of migalastat for patients with amenable mutations in the U.S.”

The U.S. market for migalastat is estimated to be approximately 25% of the global opportunity.

Migalastat is designed to selectively and reversibly bind with high affinity to the active sites of certain mutant forms of alpha-Gal A, the genotypes of which are referred to as amenable mutations. On May 30, 2016, the European Commission granted full approval for migalastat, under the trade name Galafold™, as a first line therapy for long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease (alpha-galactosidase A deficiency) and who have an amenable mutation. This EU approval may serve as the basis for regulatory approvals in more than two-thirds of the global Fabry market that is outside the U.S. Amicus has commenced the commercial launch of Galafold in Germany and is undergoing the EU country-by-country processes to launch in the majority of EU countries throughout 2016 and 2017. The Company has also initiated expanded access programs (EAP) in the EU and other territories outside the U.S. that provide this mechanism for reimbursed access prior to formal approval.

Conference Call and WebcastAmicus Therapeutics will host a conference call and audio webcast today, November 28, 2016 at 4:30 p.m. ET. Interested participants and investors may access the conference call by dialing 877-303-5859 (U.S./Canada) or 678-224-7784 (international).

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