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 bodys 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.
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
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.
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.
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.
• 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
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.
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.
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.