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Migalastat: First Global Approval

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Abstract

Migalastat (Galafold™)—a small molecule drug developed by Amicus Therapeutics that restores the activity of specific mutant forms of α-galactosidase—has been approved for the treatment of Fabry disease in the EU in patients with amenable mutations. Fabry disease is a rare disorder that results in a deficiency or absence of α-galactosidase, leading to accumulation of globotriaosylceramide in the lysosomes of various cells. This article summarizes the milestones in the development of migalastat leading to this first approval in the EU for the long-term treatment of adults and adolescents aged ≥16 years with a confirmed diagnosis of Fabry disease.

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References

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Authors and Affiliations

  1. Springer, Private Bag 65901, Mairangi Bay, 0754, Auckland, New Zealand

    Anthony Markham

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  1. Anthony Markham
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Correspondence to Anthony Markham.

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Disclosure

The preparation of this review was not supported by any external funding. During the peer review process the manufacturer of the agent under review was offered an opportunity to comment on the article. Changes resulting from any comments received were made by the author on the basis of scientific completeness and accuracy. A. Markham is a contracted employee of Adis, Springer SBM.

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This profile has been extracted and modified from the AdisInsight database. AdisInsight tracks drug development worldwide through the entire development process, from discovery, through pre-clinical and clinical studies to market launch and beyond.

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Markham, A. Migalastat: First Global Approval. Drugs 76, 1147–1152 (2016). https://doi.org/10.1007/s40265-016-0607-y

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