EMA Committee Backs First Oral Drug for Fabry Disease

Megan Brooks


April 04, 2016

The European Medicines Agency's (EMA's) Committee for Medicinal Products for Human Use has recommended in favor of marketing migalastat (Galafold, Amicus Therapeutics UK Ltd), the first oral treatment for Fabry disease.

Fabry disease is an inherited fat storage disorder caused by an X-linked deficiency of the enzyme alpha-galactosidase A, which is involved in the biodegradation of lipids. It results in the build-up of the lipid globotriaosylceramide in the vascular epithelium, heart, kidneys, cornea, and other tissues, increasing the risk for kidney failure, myocardial infarction and stroke, and other problems.

The current standard treatment for the disease is enzyme replacement therapy (ERT), which consists of an intravenous infusion of a copy of the missing enzyme. Oral migalastat may provide a more convenient treatment option, the EMA says.

The drug works differently than ERT, "acting as a 'pharmacological chaperone' which binds to the defective alpha-galactosidase A enzyme, allowing it to be transported to where its action is needed and restore its activity," the agency explains in a news release.

Migalastat is indicated only in patients with specific disease mutations known to respond to migalastat, the EMA notes.

The favorable opinion was based on the results of two phase 3 clinical trials involving about 110 patients with Fabry disease who had a genetic mutation that responds to migalastat. The drug was effective compared with placebo and to ERT in a long-term comparative study. The most common adverse effect was headache.

The Committee for Medicinal Products for Human Use opinion will now be sent to the European Commission for the adoption of a decision on marketing authorization for the European Union.


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