General Information

Galafold (Migalastat) is indicated for people with a certain inherited disease called Fabry disease. It is a rare genetic disease that may affect many parts of the body. It includes the heart, kidney, and skin. In Fabry disease, a certain enzyme in your body does not work the way it should. It leads up of a certain fatty substance in your kidneys, heart, or other organs.

This medication is used for long-term treatment. This may also decrease some symptoms caused by the disease such as:

• Diarrhea
• Abdominal cramps
• Heat intolerance
• Burning pain in the hands and feet
• Kidney or heart disease
• Skin rash
• Stroke

How does Galafold (Migalastat) work?

Galafold (Migalastat) reversibly binds to the active site of the alpha-Gal-A protein which is deficient in Fabry disease. This binding stabilizes alpha-Gal-A protein allowing its trafficking. It is from the endoplasmic reticulum into the lysosome where it exerts its action. At a higher concentration of relevant substrates, this drug dissociates from alpha-Gal-A allowing it to break down some substance. A certain variant that causes the disease may result in the production of abnormally folded and less stable forms of it. However, it retains enzymatic activity. It helps the alpha-Gal-A work better so it can remove the fatty substance.

Why use Galafold (Migalastat)?

Galafold (Migalastat) is an alpha-galactosidase-A pharmacological chaperone. It is supplied as a capsule for oral use in the strength of 150 mg. Each capsule contains Migalastat as an active ingredient. This was approved by the US Food and Drug Administration (FDA).

How to use Galafold (Migalastat)?

Galafold (Migalastat) is to be taken by mouth with a full glass of water. It is usually taken once every other day. Take each dose on an empty stomach as directed by your doctor. Do not eat at least 2 hours after taking the drug. Taking this medication with food will make it work less well. Swallow the capsule as a whole. Do not break or chew it.

The dosage is based on your medical condition and response. Don’t forget to take it at the same time on every other day.

What are the side-effects of Galafold (Migalastat)?

• Stuffy or runny nose
• Headache
• Nausea
• Fever
• Urinary tract infections (UTI)

Warnings and Precautions

• Do not increase or change the dose without consulting your doctor. Your condition may not improve any faster. It may also cause a higher risk of serious side effects.
• This is intended to take every other day. Do not take this medication 2 days in a row.
• Tell your doctor if you have any medical history, current medications, or allergies.
• Keep this medicine away from heat or moisture.