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Brand Name :
Galafold
Synonyms :
migalastat
Class :
Pharmacologic Chaperones
Brand Name :
Galafold
Synonyms :
migalastat
Class :
Pharmacologic Chaperones
Dosage Forms & Strengths
Capsule
123mg
It is indicated for adults diagnosed with Fabry disease and amenable GLA (galactosidase alpha gene) variant in vitro data
123 mg orally once on alternate days at the same time of the day
No dose adjustment is required in the case of mild to moderate renal impairment
But it is not recommended when severe end-to-end renal disease persists
Safety and efficacy are not seen in pediatrics
Refer to the adult dosing
It may enhance the risk of adverse effects when combined with Metabolic agents
It may enhance the risk of adverse effects when combined with Metabolic agents
Actions and Spectrum:
Actions
migalastat is classified as a chaperone therapy or a pharmacological chaperone. It stabilizes the mutant forms of α-Gal A enzyme, allowing it to fold correctly and reach its functional state within the lysosomes. By facilitating proper enzyme folding, migalastat helps restore α-Gal A activity, leading to a reduction in the buildup of GL-3 in various organs and tissues.
Spectrum
The spectrum of migalastat is limited to treating Fabry disease caused by specific mutations in the alpha-galactosidase A gene. It is specifically indicated for use in individuals with amenable mutations, meaning that the mutations respond to migalastat therapy. Genetic testing is typically performed to determine patients’ eligibility for migalastat treatment.
Frequency defined
>10%
Headache (35%)
Nausea (12%)
Pyrexia (12%)
Nasopharyngitis (18%)
Urinary tract infection (15%)
1-10%
Abdominal pain (9%)
Diarrhea (9%)
Epistaxis (9%)
Back pain (9%)
Cough (9%)
Black Box Warning:
None
Contraindication/Caution:
Contraindications
Cautions
Pregnancy consideration:
Insufficient data is available.
Breastfeeding warnings:
No data is available regarding the excretion of drug in breast milk.
Pregnancy category:
Category A: well-controlled and satisfactory studies show no risk to the fetus in the first or later trimester.
Category B: there was no evidence of risk to the fetus in animal studies, and there were not enough studies on pregnant women.
Category C: there was evidence of risk of adverse effects in animal reproduction studies, and no adequate evidence in human studies must take care of potential risks in pregnant women.
Category D: adequate data with sufficient evidence of human fetal risk from various platforms, but despite the potential risk, and used only in emergency cases for potential benefits.
Category X: Drugs listed in this category outweigh the risks over benefits. Hence these categories of drugs need to be avoided by pregnant women.
Category N: No data is available for the drug under this category.
Pharmacology:
migalastat binds to the mutant forms of α-Gal A, stabilizes them, and promotes their proper folding and trafficking to lysosomes. By enhancing the correct folding of the mutant enzyme, migalastat helps restore its activity, enabling it to break down GL-3 efficiently.
Pharmacodynamics:
The primary objective of migalastat therapy in Fabry disease is to reduce the accumulation of GL-3 in tissues and organs affected by the disease. This can help alleviate symptoms and slow the progression of the condition. The effects of migalastat may vary depending on the individual and the extent of GL-3 accumulation in different tissues.
Sometimes, migalastat may be combined with enzyme replacement therapy (ERT) for Fabry disease. ERT involves administering a synthetic form of the α-Gal A enzyme intravenously. migalastat can help stabilize mutant forms of the enzyme, potentially enhancing the effectiveness of ERT in individuals with amenable mutations.
Pharmacokinetics:
Absorption
Absolute bioavailability is around 75%
Peak plasma time is achieved in3 hours
Distribution
The apparent volume of distribution is 89 L (ranging from 77-133 L) at a steady state in Fabry-affected patients
Metabolism
Based on in vivo data, the drug is a substrate for (UDPGT), uridine diphosphate glucuronosyltransferase, a minor elimination pathway.
Elimination and Excretion
The drug is excreted at 77% in the urine and 20% in feces
The half-life is 4 hours
The rate of clearance is 12.5 L/hr
Administration:
migalastat is typically administered orally in the form of capsules. The specific dosing instructions and schedule may vary depending on the individual’s condition and the guidance provided by the prescribing healthcare professional.
Patient information leaflet
Generic Name: migalastat
Pronounced as: mi-GAL-a-stat
Why do we use migalastat?
migalastat treats Fabry disease in individuals with specific alpha-galactosidase A (α-Gal A) gene mutations. Fabry disease is a rare genetic disorder specified by the deficiency or dysfunction of the α-Gal A enzyme, leading to the accumulation of globotriaosylceramide (GL-3) in various tissues and organs.
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