The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended a marketing authorisation of Amicus Therapeutics’ Galafold (migalastat) to treat Fabry disease in the EU.
Galafold is the first oral treatment for Fabry disease and may provide a more convenient treatment option for patients, who are currently treated by enzyme replacement therapy (ERT).
Fabry disease is a rare genetic disorder in which patients do not have enough of an enzyme called alpha-galactosidase A.
The enzyme is responsible for the breakdown of a fatty substance called globotriaosylceramide (GL-3), EMA said.
When GL-3 is not broken down it builds up in the body’s cells, such as heart and kidney cells.
Patients with Fabry disease may have a wide range of symptoms, including severe conditions such as kidney failure, heart problems and increased risk of strokes.
The CHMP recommended granting Galafold marketing authorisation to treat patients with certain mutations of the disease which are known to be responsive to the active substance in the medicine, migalastat.
The recommendation is based on results of two Phase III clinical trials which evaluated the efficacy of Galafold, by comparing it to placebo and to ERT.
In these studies, patients taking Galafold did not experience serious side effects; the most common side effect was a headache.