FDA Accepts Migalastat New Drug Application

This morning, Amicus Therapeutics announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) submitting for filing under priority review for migalastat HCI (migalastat) for the treatment of patients with Fabry disease with amenable mutations.

Amicus submitted the NDA to the FDA halfway through December, and its intended indication makes up approximately 35% to 50% of the patient population. In July, both sides met and agreed that sufficient data evaluating the safety and efficacy of the oral precision medicine were available.

Fabry disease is a genetic lysosomal disorder resulting from a deficiency of alpha-galactosidase A (a-Gal A), the enzyme responsible for the breakdown of lipids like globotriaosylceramide (GL-3). The buildup of these fats can lead to progressive damage to multiple organs, including the skin, eyes, heart, and brain.

The NDA submission was based on clinical data from completed studies, including reduction in disease-causing substrate (GL-3), as well as the totality of data from two Phase 3 pivotal studies in treatment-naïve (FACETS) and enzyme replacement therapy (ERT) switch patients (ATTRACT).

“The FDA’s acceptance of our first Amicus NDA submission under priority review is an important step toward a potential oral precision medicine option for the Fabry disease community in the U.S.,” said John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. “With more than a decade of experience in treating patients with migalastat globally, our team at Amicus has collaborated with leading Fabry disease experts and patient organization leaders to assemble a robust NDA that emphasizes the breadth of our clinical data and experience delivering migalastat to patients. We look forward to continuing to work collaboratively with FDA to bring this oral precision medicine to patients who may be able to benefit.”

Migalastat had previously received both Orphan Drug and Fast Track designations from the FDA, and the new Priority Review status will accelerate its review time from 10 months to, ideally, six months from the day of acceptance of filing. The Prescription Drug User Fee Act (PDUFA) goal date has been set for August 13, 2018.

Under the European Commission (EC), Migalastat has already been granted full approval, under the trade name Galafold, as a first line therapy for long-term treatment of adults and adolescents ages 16 years and older with a confirmed diagnosis of Fabry disease with the amenable mutation. In the U.S., however, an estimated 3,000 people are currently diagnosed with the condition — more than in any other country.

In the aforementioned studies, no serious adverse events (AEs) had been found over the course of four years. The most common side effect in clinical trials was headache (approximately 10% of patients), and less common side effects included unspecific symptoms, like dizziness, fatigue, and nausea, but also occasionally depression.

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