How does Galafold work?
In people with Fabry disease and an amenable mutation, Galafold stabilises the faulty enzyme and aids its transport through the cell to the lysosome (a specialised fluid-filled sac found in cells that contains enzymes). Once in the lysosome, Galafold detaches from the enzyme which allows α-Gal A to breakdown GL-3.1
See the “About Fabry Disease” page for more information.
Click on the animation below to see how Galafold works, how to take it and what to do if you miss a dose or experience any side effects.
What is an amenable mutation?1
- There are many different mutations that can happen in the α-Gal A enzyme causing Fabry disease.
- Galafold works on some of these mutations, which are therefore called amenable mutations. Those mutations which don’t work with Galafold are called non-amenable.
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Enzymes are responsible for a range of jobs around the body, including recycling and removing waste products from your cells.
When our bodies are working well, this complex system functions seamlessly.
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Fabry disease means a particular enzyme in your body is misshapen or absent.
This enzyme is one of those responsible for cleaning away the waste that would otherwise build up.
Fabry disease is caused by this enzyme not working properly.2,3
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This means the waste material that should be removed starts to build up and, over time, can cause damage to your body - leading to problems including kidney failure, heart failure, stroke, hearing loss and cataracts.2,3
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Your Fabry disease is being treated with Galafold.
Galafold makes its way into your cells and binds to the misshapen enzyme…
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…which moves to the right part of the cell so that it can help it work effectively, clearing the waste products away.1
How to take Galafold?
Galafold contains the active substance migalastat.1 This medicine is used for the long-term treatment of Fabry disease in adults and adolescents aged 12 years and older who have certain specific changes in their DNA known as amenable mutations.1
Always take this medicine exactly as your doctor, pharmacist, or nurse has told you. Check with your doctor, pharmacist, or nurse if you are not sure.
- Take one capsule every other day at the same time of the day.1
- Do not take Galafold on two consecutive days.1
- Do not eat food at least 2 hours before and 2 hours after taking your medicine. This minimum 4 hours fast around taking your medicine is needed to allow your medicine to be fully absorbed. Water can be consumed during this period.1
- Swallow the capsule whole. Do not cut, crush, or chew the capsule.1
Galafold capsules are packaged in blister cards. To remove a capsule: 4
- turn the card so that the back is showing
- bend the card to help raise the oval perforated cardboard
- remove the oval perforated cardboard
- turn the card back over
- push the capsule out
What if I miss a dose?
If you forget to take your capsule at the usual time, but remember later, you can take the capsule only if it is within 12 hours of your normal dosing time. If more than 12 hours has passed, you should resume taking Galafold at the next planned dosing day and time according to your 48 hour dosing schedule. Do not take two capsules to make up for your missed dose.1
What if I take too much Galafold?
If you take more capsules than you should, then you should stop taking the medicine and contact your doctor. You may get a headache and feel dizzy.4
MyDay App
The Amicus MyDay App has been developed to help support you and help you establish a routine in taking your capsule. If you would like to use the MyDay App, it is free to download on the Apple App store and Android Play store following invitation from the healthcare professional looking after your Fabry disease.
Please read the package leaflet in the medication box. If you have any questions, please speak to your doctor or pharmacist.
Galafold Package leaflet
For more detailed information about your medicine and the current package leaflet, click here to be taken to the medicines.org.uk website.
How to remove a Galafold capsule
Click on the video below to see how to remove Galafold capsules from the pack.
Side Effects
If you get any side effects, talk to your doctor or nurse immediately. A full list of possible side effects can be found in the Patient Information Leaflet (PIL) for Galafold®.
Like all medicines, this medicine can cause side effects, although not everybody gets them. The most common side effect is headache, which may affect more than 1 in 10 people. There are also a number of common side effects, which may affect up to 1 in 10 people, including palpitations, vertigo, diarrhoea, nausea, constipation, tiredness, muscle pain, dizziness, shortness of breath and depression. See the PIL for Galafold® for the full list of side effects.
Allergic reactions
Galafold® contains 123 mg of migalastat and other ingredients. Do not take Galafold® if you are allergic to any of these ingredients:
- Pregelatinised starch (maize) and magnesium stearate
- Gelatin, titanium dioxide (E171), and indigo carmine (E132)
- Shellac, black iron oxide, and potassium hydroxide
Other treatments and Galafold®
Galafold® should not be taken if you are receiving enzyme replacement therapy.
Warnings and precautions with Galafold®
Galafold® is not for children (aged 12 years or under) weighing less than 45 kg.
Talk to your doctor before taking Galafold® if you have severely reduced kidney function as Galafold® is not recommended for patients with severe renal insufficiency (GFR less than 30 mL/min/1.73m2).
Pregnancy & breastfeeding
There is very limited experience with the use of Galafold® in pregnant women. Galafold® is not recommended for pregnant women.
If you are pregnant, think you may be pregnant, or are planning to have a baby, ask your doctor for advice before taking this medicine.
Women who could become pregnant should use effective birth control while taking Galafold®.
Do not take this medicine if you are breast-feeding, until you have spoken with your doctor, pharmacist, or nurse. It is not yet known whether this medicine passes into breast milk. Your doctor will decide whether to stop breast-feeding or temporarily stop taking Galafold®, considering the benefit of breast-feeding to the baby and the benefit of taking Galafold® to the mother.
Fertility
Fertility in men:
It is not yet known if this medicine affects fertility in men. The effects of Galafold® on fertility in humans have not been studied.
Fertility in women:
It is not yet known if this medicine affects fertility in women. If you are planning to have a baby, ask your doctor, pharmacist, or nurse for advice.
References
- Summary of product characteristics. Galafold 123 mg hard capsule. Amicus Therapeutics Ltd. Available at: https://www.medicines.org.uk/emc/product/10934/smpc
- Germain DP. Fabry disease. Orphanet J Rare Dis. 2010;5:30. doi:10.1186/1750-1172-5-30
- Hoffmann B, Mayatepek E. Fabry disease-often seen, rarely diagnosed. Dtsch Arztebl Int. 2009;106(26):440-447.
- Package leaflet: Information for the patient. Galafold 123 mg hard capsules. Amicus Therapeutics Ltd. Available at: https://www.medicines.org.uk/emc/product/10934/pil
