Migalastat set for approval as first oral therapy for Fabry disease

α-galactosidase A enzyme

The European Medicines Agency (EMA) has recommended the approval of migalastat for the treatment of the rare genetic disorder Fabry disease.

If approved, the drug will be the first oral therapy available for the disease, which is currently treated with intravenous enzyme-replacement therapy (ERT).

Fabry disease is a lysosomal storage disorder caused by a deficiency of the α-galactosidase A enzyme, resulting in accumulation of the lipid GL-3 in multiple tissues. This can lead to neuropathic pain, renal failure, cardiovascular disease and an increased risk of stroke. Migalastat functions by binding to the defective enzyme, allowing it to be chaperoned to the lysosome where enzyme activity is restored.

The EMA’s decision was based on data from two phase III clinical trials involving 110 patients, which compared migalastat with placebo and ERT. These trials showed that cardiac and renal function were improved at 18 months in patients who had previously been treated with ERT, and at 30 months in ERT-naive patients. Long-term studies are ongoing.

There are more than 800 mutations known to cause Fabry disease. Of these, 269 are responsive to treatment with migalastat and account for 35–50% of disease cases. If approved, the treatment could be prescribed first-line to patients aged 16 years and over who have one of these mutations.

Migalastat’s manufacturer, Amicus, says it expects the European Commission to decide whether to grant the drug marketing authorisation by the end of the second quarter.

Last updated
The Pharmaceutical Journal, Migalastat set for approval as first oral therapy for Fabry disease;Online:DOI:10.1211/PJ.2016.20200966

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