FDA approves first oral therapy for rare Fabry disease

pharmafile | August 13, 2018 | News story | Sales and Marketing FDA, Fabry disease, US, pharma 

Amicus has revealed that its therapy Galafold (migalastat) has been awarded approval by the FDA in the treatment of adult patients with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data.

A rare genetic disorder, Fabry disease is characterised by an enzyme deficiency which can lead to the development of progressive kidney disease, cardiac hypertrophy, arrhythmias, stroke and early death. The condition affects an estimated 3,000 in the US, with more than half of this number thought to be going without treatment.

The US regulator’s decision was made under accelerated approval and was based on six months of Phase 3 data generated though the treatment of 45 treatment-naïve patients with the condition, in which the drug demonstrated a greater reduction in globotriaosylceramide (GL-3) in blood vessels of the kidneys compared to placebo.

“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,” commented Jack Johnson, Founder and Executive Director, Fabry Support & Information Group. “With the FDA approval of Galafold, certain members of the US 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.”

John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, also remarked: “This FDA approval of Galafold is a transformative moment for people in the US 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 US 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 US leadership team, we are poised to make Galafold available to as many appropriate patients as possible.”

Matt Fellows

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