Vijayaganesh Kasinathan
Written & Compiled by Pharmacist Vijayaganesh Kasinathan, B.Pharm, M.Pharm
Last Updated on Aug 23, 2018
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General Information on Migalastat

Generic Name : Migalastat

Pronunciation : mi-GAL-a-stat

Latest prescription information about Migalastat. Learn how to pronounce the drug's name, its indications, dosage, how to take, when to take, when not to take, side effects, special precautions, its storage instructions and warnings if any when taken during pregnancy. Also listed are the International and Indian trade name(s) of the drug and its price list.
Therapeutic Classification : Orphan Drug

Trade Names/Brand Names of Migalastat


Why is Migalastat Prescribed? (Indications)

Migalastat is prescribed to treat Fabry disease in adult patients. Fabry disease is a rare, genetic and an inherited disorder that occurs due to an accumulation or bulid-up of a type of fat known as globotriaosylceramide (GL-3) in the blood vessels, nerves, heart, the kidneys, and other organs.

Migalastat is used only after the confirmation of an alpha-galactosidase enzyme deficiency or a permanent gene alteration in the alpha-galactosidase gene A (GLA) located on the X chromosome.

Patients with Fabry disease may develop abnormal heart rhythm, enlargement of the heart, progressive kidney disease, stroke and early death.

Migalastat works by increasing the activity of the body’s deficient enzyme rather than acting as an enzyme replacement therapy. Patients treated with migalastat over six months showed a more significant reduction in globotriaosylceramide (GL-3) in the blood vessels of the kidneys. 

When should Migalastat not to be taken? (Contraindications)

Migalastat should not be used in patients with-

• If there is allergy to migalastat
• End-stage renal disease
• Infertility treatment (only in males)
• Children below 18 years of age

What is the dosage of Migalastat?

• The recommended adult dose is one capsule of Migalastat 123 mg taken on alternate days.

• Migalastat is mainly excreted by the kidneys; therefore no dose adjustment is required in patients with liver disease and mild to moderate kidney disease.

Missed a Dose?

If a dose of migalastat is missed, take the missed dose within 12 hours. If the gap is more than 12 hours avoid taking the missed dose and continue with the next dose according to the scheduled time.

How should Migalastat be taken?

• Migalastat is available in capsule form should be taken by mouth every other day mainly at the same time of each day.

• Take migalastat capsules only on an empty stomach. Avoid eating at least 2 hours before and 2 hours after while taking migalastat capsules.

• Patients are allowed to drink water during this time gap of 4 hours.

• Never take migalastat capsules continuously for two consecutive days.

• Do not chew, crush, cut or open the capsule contents but swallow the capsule as a whole with sufficient quantity of water.

What are the warnings and precautions for Migalastat?

• Monitor patients for their heart and kidney function along with other biochemical markers every six months if migalastat has been prescribed or patient is switched to migalastat from other medications.

• Women of reproductive potential who are taking migalastat should be advised to use an effective contraceptive measure to prevent pregnancy.

• Caution is required while giving migalastat capsules to pregnant and breastfeeding women to avoid harm to the fetus or the baby particularly when large doses are prescribed.

What are the side effects of Migalastat?

•  Gastrointestinal: Nausea, stomach pain, loose stools or urgency to defecate, indigestion, dry mouth, constipation
•  Nervous system: Headache, light-headedness, decreased sense of touch or sensation, abnormal sensation of numbness or tingling, depressive illness, weakness
•  Respiratory: Nosebleed, difficulty in breathing, cough, infection of the pharynx, stuffy or a runny nose, sore throat
•  Musculoskeletal: Muscle pain, torticollis, muscle cramps, back pain, pain in the extremities such as hands, wrist, or foot
•  Others: Abnormal heart rhythm, fever, rashes or itching, presence of protein in the urine, weight gain, abnormal levels of creatine phosphokinase in the blood

What are the other precautions for Migalastat?

• Patients should be treated with general medical care in case of an overdose of migalastat which may generally lead to a severe headache and dizziness.

• Never use migalastat capsules for conditions other than what it is prescribed for.

• Do not recommend migalastat capsules to another person with similar symptoms unless otherwise approved by the doctor.

What are the Drug Interactions of Migalastat?

• Inform the doctor about the prescription, Over-The-Counter drugs, vitamins, or herbal supplements you are taking or planned to take.

• This helps to avoid life-threatening side effects that occur due to the drug interactions.

What are the storage conditions for Migalastat?

• Store the migalastat capsules at room temperature between 20°C and 25°C.

• Keep the capsules in the blister card they come in.

• Keep migalastat capsules out of the reach of the children.

Last Updated : 8/23/2018

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