drugs

Galafold - Migalastat

What is Galafold - Migalastat used for?

Galafold is a medicine used to treat patients aged 16 or over with Fabry disease. It is a rare hereditary disease in which patients present various mutations (variations) of the gene responsible for producing an enzyme called alpha-galactosidase A, which normally breaks down a fatty substance called globotriaosilceramide (GL-3). In patients with Fabry disease, this enzyme is not functioning properly. As a result, GL-3 cannot be broken down and accumulates in various cells of the body, including heart and kidney cells.

Because the number of patients with Fabry disease is small, the disease is considered 'rare' and Galafold was designated an 'orphan medicine' (a medicine used in rare diseases) on 22 May 2006.

Galafold contains the active ingredient migalastat.

How is Galafold - Migalastat used?

Galafold can only be obtained with a prescription and treatment should be started and monitored by a doctor experienced in the diagnosis and treatment of Fabry disease.

Galafold is available as capsules (123 mg). The recommended dose of Galafold is one capsule every other day, taken orally at least 2 hours before or after food consumption.

Galafold should only be used in patients who have certain mutations of the alpha-galactosidase A gene. For more information, see the summary of product characteristics (also part of the EPAR).

How does Galafold - Migalastat work?

Galafold contains the active ingredient migalastat, which binds to certain unstable forms of alfagalactosidase A, stabilizing the enzyme. This allows the enzyme to be transported to areas of the cell where it can break down the GL-3.

What benefit has Galafold - Migalastat shown during the studies?

Galafold was analyzed in two main studies involving a total of 127 patients with Fabry disease.

The first study, which compared Galafold with placebo (a dummy treatment) in 67 patients, assessed the percentage of patients who responded to treatment (defined as a reduction of at least 50% of GL-3 deposits in the kidney) . In general, Galafold was not more effective than placebo in reducing GL-3 deposits; however, other analyzes that included only patients with those genetic mutations that can be treated with Galafold showed that patients responded better to Galafold than to placebo after 6 months of treatment.

The second study of 60 patients compared Galafold with the agalsidase alfa and agalsidase beta substances, two substitutive treatments of the missing enzyme. The main measure of effectiveness was the change in patients' renal function after 18 months of treatment. In this study, Galafold was as effective as enzyme replacement therapy in stabilizing patients' renal function.

What is the risk associated with Galafold - Migalastat?

The most common side effect of Galafold (which may affect about 1 in 10 people) is headache.

For the full list of restrictions and side effects reported with Galafold, see the package leaflet.

Why has Galafold - Migalastat been approved?

The Agency's Committee for Medicinal Products for Human Use (CHMP) decided that Galafold's benefits are greater than its risks and recommended that it be approved for use in the EU. The committee noted that Galafold has been studied in a limited number of patients, however the available evidence is considered sufficient for such a rare disease. The CHMP also considered that Galafold is taken orally and this could be an advantage over other authorized treatments such as enzyme replacement therapy, which are given by infusion (drip) into a vein. As for safety, Galafold was well tolerated.

What measures are being taken to ensure the safe and effective use of Galafold - Migalastat?

A risk management plan has been developed to ensure that Galafold is used as safely as possible. Based on this plan, safety information has been included in the summary of product characteristics and the package leaflet for Galafold, including the appropriate precautions to be followed by healthcare professionals and patients.

More information on Galafold - Migalastat

For the full EPAL of Galafold, consult the Agency's website: ema.europa.eu/Find medicine / Human medicines / European public assessment reports. For more information on Galafold therapy, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.

The summary of the opinion of the Committee for Orphan Medicinal Products related to Galafold is available on the Agency's website: ema.europa.eu/Find medicine / Human medicines / Rare disease designation.