Fabry Disease Treatment Approved in Japan

Article

In February, the U.S. FDA accepted the New Drug Application for migalastat, a potential Fabry disease therapy in development by Amicus. This morning, it was announced that it was approved in Japan.

The Fabry disease community continues to make major strides in 2018.

In February, the U.S. Food and Drug Administration (FDA) accepted the New Drug Application (NDA) for migalastat, a potential Fabry disease therapy in development by Amicus Therapeutics. This morning, it was announced that the first-in-class chaperone therapy was approved in Japan for patients aged 16 years and older with an amenable mutation.

Japan’s Ministry of Health, Labour and Welfare (MHLW) has given the go-ahead to Galafold capsules 123 mg (migalastat), and Amicus will now proceed with pricing and reimbursement processes, and anticipates launching the drug in Japan in the coming months.

The approval was based on clinical data from two pivotal Phase 3 studies — FACETS and ATTRACT – which evaluated treatment naïve patients and patients who had previously received enzyme replacement therapy (ERT), respectively.

“Japan is very important to our patient-focused vision to provide Galafold to Fabry patients with amenable mutations throughout the world as soon as possible,” said John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. in a press release. “And now that Amicus has established a strong presence in Japan, we hope that the upcoming Galafold launch will be the first of many future opportunities to deliver new medicines for people living with rare metabolic diseases in Japan.”

Approximately 850 people in Japan are currently known to be living with Fabry disease. Migalastat has exhibited the ability to clear the accumulated globotriacylceramide (GL-3) in the lysosome and stabilize the body’s dysfunctional enzyme. It is believed that a significant portion of patients have amenable mutations, or mutations that are responsive to therapy with migalastat based on a proprietary in vitro assay.

The Galafold Amenability Assay was used to classify more than 1,000 known GLA mutations as either “amenable” or “not amenable” to treatment with migalastat.

“As a principal investigator in the Galafold pivotal studies with extensive experience treating Fabry disease, it can be said that significant unmet need remains,” said Professor Toya Ohashi, Jikei University. “Galafold has a unique mechanism of action that has demonstrated compelling results in naïve and treatment-experienced Fabry patients who have amenable mutations. This differentiated treatment option is good news for the many Fabry patients in Japan who have an amenable mutation.”

In the FACETS and ATTRACT studies, no serious adverse events (AEs) had been observed over a 4 year span. The most common side effect in clinical trials was headache (approximately 10% of patients), and less common side effects were unspecific symptoms including: dizziness, fatigue, and nausea, and occasionally depression.

In the U.S., approximately 3,000 people are currently known to be living with Fabry disease. Migalastat has previously received both Orphan Drug and Fast Track designations from the FDA, and its recent Priority Review status will expedite its review time from 10 months to six. The Prescription Drug User Fee Act (PDUFA) goal date has been set for August 13, 2018.

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