What condition does Migalastat treat?

Migalastat is used for people with a certain inherited disease called Fabry disease. It is a rare genetic disease that may affect many parts of the body including the heart, kidney, and skin. In Fabry disease, a certain enzyme in your body does not work the way it should. It leads up to 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:

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

Migalastat is an alpha-galactosidase-A pharmacological chaperone. It is supplied as a capsule for oral use. This was approved by the US Food and Drug Administration (FDA).

How does Migalastat work?

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.

What is the recommended dosage for Migalastat?

This medicine is to be taken by mouth with a full glass of water. the medicine 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 every other day. Do not increase your dose or use this drug more often or for longer than prescribed. Your risk of serious side effects will increase and your condition will not improve any faster.

What are the side effects of Migalastat?

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

What are the precautions in taking Migalastat?

  • During pregnancy, this medicine should be used only when needed. Ask about reliable forms of birth control while using this medication. If you become pregnant, talk to your doctor right away about the risks and benefits of this medication.
  • This is intended to take every other day. Do not take this medication 2 days in a row.
  • Do not use machinery, drive, or do anything that needs alertness until you can do it safely. This drug may make you dizzy.
  • It is unknown if this medication passes into breast milk. Consult your doctor before breastfeeding.
  • Tell your doctor if you have any medical history, current medications, or allergies.
  • Keep this medicine away from heat or moisture.