FDA Approves Galafold™ (migalastat) for the Treatment of Certain Adult Patients with Fabry Disease

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CRANBURY, N.J., Aug. 10, 2018 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval of Galafold™ (migalastat) 123 mg capsules. Galafold is an oral, precision medicine for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene () variant based on assay data.

Galafold was approved under the Subpart H Accelerated Approval pathway based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. As a condition of accelerated approval, Amicus Therapeutics will continue to study Galafold in a confirmatory Phase 4 program. Galafold is a capsule taken once every other day, at the same time of day. The FDA has approved Galafold for 348 amenable variants. Galafold is the first oral medicine for Fabry disease, and the first new therapy approved to treat Fabry disease in the United States in more than 15 years. 

John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc., stated, “This FDA approval of Galafold is a transformative moment for people in the U.S. living with Fabry disease, as it gives adult patients with amenable variants a new treatment option for the first time in more than 15 years. The Fabry disease community has had an active voice in every stage of development of this medicine. We are grateful to this wonderful and passionate community, particularly the patients and physicians who have made this research possible through their participation in the clinical trials, as well as to the U.S. regulators and our ever-persistent and dedicated Amicus team. This achievement highlights our company’s mission, which is focused on transforming the lives of people living with rare diseases. With our new and highly motivated U.S. leadership team, we are poised to make Galafold available to as many appropriate patients as possible.”

Fabry disease is a rare, progressive genetic disorder characterized by a defective gene () that causes an enzyme deficiency. This enzyme is responsible for breaking down disease substrate that, when deficient in patients with Fabry disease, builds up in the kidneys, which is one of the organ systems impacted by Fabry disease. In the U.S., it is estimated that more than 3,000 people are living with Fabry disease, and an estimated more than 50 percent of these diagnosed patients are currently untreated. Globally, it is estimated that 35 to 50 percent of Fabry disease patients may have an amenable variant.  

“People with rare diseases often have limited treatment options, so research leading to new therapies is critical. As a physician and researcher, I treated the first patient ever to receive Galafold in a clinical trial. Today, many years later, I am pleased that more patients will have access to this precision medicine,” said Raphael Schiffmann, M.D., M.H.Sc., Director, Baylor Scott & White Research Institute, Kimberly H. Courtwright and Joseph W. Summers Institute of Metabolic Disease – Dallas.

The FDA approval was based on data from a Phase 3 pivotal Fabry disease study in treatment-naïve patients (, or FACETS), which demonstrated the efficacy of Galafold, including a reduction in the amount of damaging disease substrate accumulated in the kidney capillaries. The most common side effects related to Galafold were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia.

“Today is a long-awaited day of celebration for all of us living with and advocating for people with Fabry disease, especially those who have participated in the development of Galafold in the United States,” said Jack Johnson, Founder and Executive Director, Fabry Support & Information Group. “With the FDA approval of Galafold, certain members of the U.S. Fabry disease patient community finally have a second treatment option. Through their unwavering commitment and scientific innovation, Amicus has provided a much-needed new treatment option for many Fabry patients.”

Amicus Therapeutics will launch Galafold immediately, and will begin shipping to a limited distribution network in the coming week. Amicus Assist, a service that will provide product assistance and support to patients to help gain access to Amicus Therapeutics’ medications, will also be initiated immediately; for more information please call +1-833-AMICUS-A (+1-833-264-2872). With today’s approval, patients in 20 countries worldwide have reimbursable access to Galafold. Galafold is approved in Australia, Canada, European Union, Israel, Japan, South Korea, Switzerland and the U.S. Galafold is pending approval in Taiwan.

An audio webcast can also be accessed via the Investors section of the Amicus Therapeutics corporate website at , and will be archived for 30 days. Web participants are encouraged to go to the website 15 minutes prior to the start of the call to register, download and install any necessary software. A telephonic replay of the call will be available for seven days beginning at 12:00 p.m. ET on August 13, 2018. Access numbers for this replay are +1-855-859-2056 (U.S./Canada) and +1-404-537-3406 (international); conference ID: 9782489.

This indication is approved under accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

It is not known if Galafold is present in human milk. Therefore, the developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for Galafold and any potential adverse effects on the breastfed child from Galafold or from the underlying maternal condition.

Galafold is not recommended for use in patients with severe renal impairment or end-stage renal disease requiring dialysis.

The safety and effectiveness of Galafold have not been established in pediatric patients.

To report Suspected Adverse Reactions, contact Amicus Therapeutics at 1-877-4AMICUS or FDA at 1-800-FDA-1088 or .

For additional information about Galafold, including the full U.S. Prescribing Information, please visit .

A video accompanying this announcement is available at 

Logos accompanying this announcement are available at  and 

A PDF accompanying this announcement is available at 

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Globe Newswire: 21:05 GMT Friday 10th August 2018

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