Migalastat hydrochloride

Migalastat Hydrochloride is a pharmaceutical drug used in the treatment of Fabry disease, a rare genetic disorder. It is marketed under the brand name Galafold by Amicus Therapeutics.

Overview

Migalastat Hydrochloride is an oral medication that works by stabilizing the body's own dysfunctional enzyme, alpha-galactosidase A (alpha-GAL), which is responsible for breaking down a specific type of fat called globotriaosylceramide (GL-3). In patients with Fabry disease, the alpha-GAL enzyme is either absent or not working properly, leading to the accumulation of GL-3 in the body's cells, which can cause a variety of symptoms and complications.

Medical Uses

Migalastat Hydrochloride is used for the long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease and who have an amenable mutation. It is not effective in all patients with Fabry disease, as its efficacy depends on the specific genetic mutation present in the patient.

Side Effects

Common side effects of Migalastat Hydrochloride include headache, nasopharyngitis, urinary tract infection, nausea and pyrexia. Serious side effects may include heart and kidney problems.

Pharmacology

Migalastat Hydrochloride is a small molecule that binds to and stabilizes specific mutant forms of the alpha-GAL enzyme, allowing it to break down GL-3. It is taken orally and is absorbed rapidly, with peak plasma concentrations reached within 3 hours.

History

Migalastat Hydrochloride was developed by Amicus Therapeutics and received its first approval in 2016 from the European Medicines Agency (EMA). It was later approved by the Food and Drug Administration (FDA) in the United States in 2018.

See Also

• Fabry disease
• Amicus Therapeutics
• European Medicines Agency
• Food and Drug Administration