Migalastat
Fabry Disease
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Drug Status
Government Approvals
US(FDA), UK(BNF)
WHO Essential Medicine
NO
Known Teratogen
No
Pharmaceutical Class
NA
Controlled Drug Substance
No
Migalastat
Summary
Migalastat is used to treat adults with Fabry disease who have a specific genetic variant that responds to the drug.
Migalastat works by stabilizing the alpha-galactosidase A enzyme. This allows the enzyme to function properly and break down certain substances in the body, reducing their accumulation.
The usual dose for adults is 123 mg taken orally every other day. It's important to avoid food and caffeine at least 2 hours before and after taking the medication to ensure proper absorption.
Common side effects of Migalastat include headache, nasopharyngitis, urinary tract infection, nausea, and pyrexia. A serious adverse effect reported is angioedema which requires immediate medical attention.
Migalastat is not recommended for patients with severe renal impairment or end-stage renal disease requiring dialysis. It should not be used in patients with non-amenable mutations. Also, patients should avoid caffeine around the time of dosing to ensure efficacy.
Indications and Purpose
How does Migalastat work?
Migalastat acts as a pharmacological chaperone, binding to the active site of the alpha-galactosidase A enzyme. This binding stabilizes the enzyme, allowing it to be properly trafficked to the lysosome, where it can break down harmful substances that accumulate in Fabry disease.
Is Migalastat effective?
Migalastat has been shown to effectively reduce kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate in patients with Fabry disease with amenable variants. Clinical trials demonstrated its efficacy in stabilizing renal function and reducing left ventricular mass index (LVMi) over long-term use.
What is Migalastat?
Migalastat is used to treat adults with Fabry disease who have a specific genetic variant that responds to the drug. It works by stabilizing the alpha-galactosidase A enzyme, allowing it to function properly in breaking down certain substances in the body, thereby reducing their accumulation.
Directions for Use
For how long do I take Migalastat?
Migalastat is intended for long-term use in the treatment of Fabry disease, as it is a chronic condition. The duration of use should be determined by a healthcare provider based on individual patient needs and response to treatment.
How do I take Migalastat?
Take Migalastat 123 mg orally every other day at the same time. Swallow the capsule whole on an empty stomach. Avoid food and caffeine at least 2 hours before and after taking the medication to ensure proper absorption.
How should I store Migalastat?
Store Migalastat at room temperature between 68°F to 77°F (20°C to 25°C). Keep the capsules in their original packaging to protect them from moisture. Ensure they are kept out of reach of children.
What is the usual dose of Migalastat?
The usual dose for adults is 123 mg taken orally every other day. The safety and effectiveness of Migalastat in children have not been established, so there is no recommended dose for pediatric patients.
Warnings and Precautions
Can I take Migalastat with other prescription drugs?
Co-administration with caffeine significantly reduces Migalastat's absorption, potentially decreasing its efficacy. Patients should avoid caffeine at least 2 hours before and after taking Migalastat. No other significant drug interactions are noted.
Can Migalastat be taken safely while breastfeeding?
There is no data on the presence of Migalastat in human milk, but it is present in the milk of lactating rats. Breastfeeding mothers should consider the benefits of breastfeeding along with the need for Migalastat and potential effects on the infant.
Can Migalastat be taken safely while pregnant?
There is limited data on the use of Migalastat during pregnancy. Animal studies showed no adverse developmental effects. Pregnant women should consult their healthcare provider to weigh the benefits and risks of using Migalastat.
Is Migalastat safe for the elderly?
Clinical trials did not include enough patients aged 65 and older to determine if they respond differently from younger patients. However, no dosage adjustment is required based on age. Elderly patients should use Migalastat under the guidance of a healthcare provider.
Who should avoid taking Migalastat?
Migalastat is not recommended for patients with severe renal impairment or end-stage renal disease requiring dialysis. It should not be used in patients with non-amenable mutations. Patients should avoid caffeine around the time of dosing to ensure efficacy.
