Medically reviewed by Drugs.com. Last updated on Oct 19, 2023.
Uses for migalastat
Migalastat is used to treat adults with Fabry disease who have a certain genetic change (variant) in an enzyme called galactosidase alpha (GLA). Your doctor will test for this GLA gene variant.
This medicine is available only with your doctor's prescription.
Before using migalastat
In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For this medicine, the following should be considered:
Tell your doctor if you have ever had any unusual or allergic reaction to this medicine or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.
Appropriate studies have not been performed on the relationship of age to the effects of migalastat in the pediatric population. Safety and efficacy have not been established.
Appropriate studies performed to date have not demonstrated geriatric-specific problems that would limit the usefulness of migalastat in the elderly.
There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.
Interactions with Medicines
Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. Tell your healthcare professional if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicine.
Interactions with Food/Tobacco/Alcohol
Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. The following interactions have been selected on the basis of their potential significance and are not necessarily all-inclusive.
Using this medicine with any of the following is usually not recommended, but may be unavoidable in some cases. If used together, your doctor may change the dose or how often you use this medicine, or give you special instructions about the use of food, alcohol, or tobacco.
Other Medical Problems
The presence of other medical problems may affect the use of this medicine. Make sure you tell your doctor if you have any other medical problems, especially:
- Kidney disease, severe (eg, end-stage kidney disease)—Use is not recommended in patients with this condition.
Proper use of migalastat
Take this medicine only as directed by your doctor. Do not take more of it, do not take it more often, and do not take it for a longer time than your doctor ordered.
This medicine comes with a patient information leaflet and patient instructions. Read and follow these instructions carefully. Ask your doctor if you have any questions.
Swallow the capsule whole. Do not cut, crush, or chew it.
Take this medicine on an empty stomach. Do not eat food or drink any product that contains caffeine at least 2 hours before and 2 hours after (total of at least 4 hours) using this medicine. You may drink water (plain, flavored, or sweetened), fruit juices without pulp, and caffeine-free carbonated beverages during this time when you cannot eat.
Do not take this medicine 2 days in a row.
The dose of this medicine will be different for different patients. Follow your doctor's orders or the directions on the label. The following information includes only the average doses of this medicine. If your dose is different, do not change it unless your doctor tells you to do so.
The amount of medicine that you take depends on the strength of the medicine. Also, the number of doses you take each day, the time allowed between doses, and the length of time you take the medicine depend on the medical problem for which you are using the medicine.
- For oral dosage form (capsules):
- For Fabry disease:
- Adults—123 milligrams (mg) once every other day at the same time of day.
- Children—Use and dose must be determined by your doctor.
- For Fabry disease:
If you miss a dose of this medicine, take it as soon as possible. However, if it is almost time for your next dose, skip the missed dose and go back to your regular dosing schedule. Do not double doses.
If you miss a dose and it is 12 hours or less from your normal dosing schedule, take the missed dose as soon as possible. If you miss a dose and it is more than 12 hours from your regular dosing schedule, skip the missed dose and take your next dose at the regular time.
Store the medicine in a closed container at room temperature, away from heat, moisture, and direct light. Keep from freezing.
Keep out of the reach of children.
Do not keep outdated medicine or medicine no longer needed.
Ask your healthcare professional how you should dispose of any medicine you do not use.
Precautions while using migalastat
It is very important that your doctor check your progress at regular visits to make sure that this medicine is working properly and to check for unwanted effects.
Side Effects of migalastat
Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.
Check with your doctor immediately if any of the following side effects occur:
- Bladder pain
- bloody or cloudy urine
- difficult, burning, or painful urination
- frequent urge to urinate
- lower back or side pain
Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:
- Back pain
- muscle aches
- sore throat
- stomach pain
- stuffy or runny nose
- unusual tiredness or weakness
Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.
Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.
Commonly used brand name(s)
In the U.S.
Available Dosage Forms:
Therapeutic Class: Endocrine-Metabolic Agent
Frequently asked questions
More about migalastat
- Check interactions
- Compare alternatives
- Side effects
- Dosage information
- During pregnancy
- Drug class: miscellaneous metabolic agents
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