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Amicus plans to submit NDA to FDA for migalastat for fabry disease

Published 12 July 2017

Amicus Therapeutics has revealed its plans to file a new drug application (NDA) with the US FDA for the oral precision medicine migalastat for Fabry disease in the fourth quarter of the year.

Based on a series of discussions with and written communication received from the FDA, the Agency has informed Amicus that it may now submit an NDA for migalastat.

Amicus is preparing the NDA submission under Subpart H, which provides for accelerated approval. Amicus intends to base its NDA on existing data, including reduction in disease-causing substrate (GL-3), as well as the totality of data from completed clinical studies.

Progressive accumulation of GL-3 is believed to lead to the morbidity and mortality of Fabry disease, including pain, kidney failure, heart disease and stroke. An additional Phase 3 study previously requested by the Agency to assess Gastrointestinal (GI) symptoms is no longer required prior to an NDA submission.

"This guidance from the FDA marks a tremendous step forward for thousands of people living with Fabry disease in the United States," stated John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics. "We are moving ahead expeditiously with our NDA submission and accelerating the U.S. pathway for migalastat. Today is a seminal moment in the development of migalastat and a testament to the dedication and perseverance of the patients, physicians and employees who have worked so hard on the development of this precision medicine."

Jay Barth, M.D., Chief Medical Officer of Amicus Therapeutics, stated, "The data from our clinical trials, including the two largest pivotal studies ever completed in Fabry disease, have already supported approvals for migalastat in the EU, Israel and Switzerland, as well as our pending regulatory submissions in Japan, Canada and Australia.

“The FDA's willingness to review migalastat data reflects what we believe is the gold standard in science-based, data-driven, patient-centric therapeutic development. We believe that we have a robust data package for this NDA submission, and we look forward to advancing toward a planned pathway for U.S. approval for migalastat."

An estimated 3,000 people in the U.S. are currently diagnosed with Fabry disease. The U.S. represents the single largest geography for Amicus to positively impact the lives of people with Fabry who have amenable mutations.

"I am very pleased that Amicus plans to submit the NDA for migalastat in the fourth quarter," said Jack Johnson, Founder and Executive Director of the Fabry Support & Information Group (FSIG).

"With significant unmet needs and a lack of treatment choices for people living with Fabry disease in the U.S., we may be one step closer to a new oral therapy. Amicus has been a true partner for the Fabry community for more than a decade, and I look forward to potentially having a new oral precision medicine available to patients with amenable mutations in the U.S."

Migalastat is an oral precision medicine intended to treat Fabry disease in patients who have amenable genetic mutations. Migalastat works by stabilizing the body's own dysfunctional enzyme, so it can clear the accumulated disease substrate in patients who have amenable mutations.

An amenable mutation is one that is responsive to therapy with migalastat based on a proprietary in vitro assay. Amicus estimates that 35%-50% of Fabry patients globally may have amenable genetic mutations.

The European Commission (EC) has 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 and who have an amenable mutation.

Marketing applications have also been approved in several countries outside the EU, including Switzerland and Israel. Regulatory submissions are under review in additional countries including Japan, Canada and Australia.

Source: Company Press Release