Cranbury-based Amicus Therapeutics 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 (GLA) variant based on in vitro assay data.
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 GLA 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 (GLA) 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 GLA 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.
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