Galafold
Fabry Disease, Fabry Disease
Treatment
3 FDA approvals
0 Active Studies for Galafold
Treatment for
Fabry Disease
What is Galafold
Migalastat
The Generic name of this drug
Treatment Summary
Fabry disease is a rare, genetic disorder caused by a missing or defective enzyme called alpha-Gal A. This enzyme is responsible for breaking down glycosphingolipid in the body, and when it is not present, the substance builds up in the blood vessels, kidneys, nerves, heart and other organs. It is estimated that over 3,000 people in the US have Fabry disease, with more than half remaining untreated.Migalastat, sold under the brand name Galafold by Amicus Therapeutics, is an oral medication used to treat Fabry disease in adults with a certain type of
Galafold
is the brand name
Galafold Overview & Background
Brand Name
Generic Name
First FDA Approval
How many FDA approvals?
Galafold
Migalastat
2018
1
Approved as Treatment by the FDA
Migalastat, otherwise known as Galafold, is approved by the FDA for 3 uses which include Fabry's Disease and Fabry Disease .
Fabry's Disease
Fabry Disease
Fabry Disease
Effectiveness
How Galafold Affects Patients
Clinical trials have shown that treatment with migalastat generally increases the levels of a specific enzyme (alpha-Gal A) in white blood cells, skin, and kidneys in most patients. For people whose GLA gene is affected, this treatment also tends to reduce levels of the chemical GL-3 in both urine and the tiny blood vessels in the kidneys.
How Galafold works in the body
Fabry disease is a genetic condition that affects both men and women. It is caused by mutations in the gene that makes the enzyme alpha-galactosidase A. This enzyme helps the body break down certain fats. When it's not functioning properly, these fats build up in the body and cause damage to organs. Migalastat is a drug that works to reduce the effects of Fabry disease. It binds to certain mutated forms of alpha-galactosidase A in the body and helps it move to the lysosomes, which are tiny sacs in cells that help break down complex molecules. Once in
When to interrupt dosage
The recommended dosage of Galafold is founded on the diagnosed condition. The amount of dosage varies, depending on the technique of administration outlined in the table beneath.
Condition
Dosage
Administration
Fabry Disease
123.0 mg,
, Oral, Capsule, Capsule - Oral
Fabry Disease
123.0 mg,
, Oral, Capsule, Capsule - Oral
Warnings
There are 20 known major drug interactions with Galafold.
Common Galafold Drug Interactions
Drug Name
Risk Level
Description
Aclidinium
Minor
Migalastat may decrease the excretion rate of Aclidinium which could result in a higher serum level.
Acrivastine
Minor
Migalastat may decrease the excretion rate of Acrivastine which could result in a higher serum level.
Albutrepenonacog alfa
Minor
Migalastat may decrease the excretion rate of Albutrepenonacog alfa which could result in a higher serum level.
Almasilate
Minor
Migalastat may decrease the excretion rate of Almasilate which could result in a higher serum level.
Alogliptin
Minor
Migalastat may decrease the excretion rate of Alogliptin which could result in a higher serum level.
Galafold Toxicity & Overdose Risk
Migalastat's most common side effects are headache, cold-like symptoms, urinary tract infection, nausea, and fever. If an overdose occurs, medical help should be sought. Headache and dizziness are the most common reactions to taking doses of up to 1250mg and 2000mg, respectively.
Galafold Novel Uses: Which Conditions Have a Clinical Trial Featuring Galafold?
Currently, there are zero active trials evaluating the potential of Galafold in providing relief from Fabry Disease.
Condition
Clinical Trials
Trial Phases
Fabry Disease
0 Actively Recruiting
Fabry Disease
0 Actively Recruiting
Patient Q&A Section about galafold
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Does Galafold work?
"The key finding of the study was that Galafold is as effective as ERT in stabilizing kidney function after 18 months of treatment."
Answered by AI
Who makes Galafold?
", Amicus Therapeutics announced that the FDA had granted accelerated approval to Galafold™. Galafold™ is a new treatment for Fabry disease, and is taken as a capsule."
Answered by AI
What is Fabry's disease?
"Fabry disease is an inherited disorder caused by the absence of activity of the lysosomal enzyme, α-galactosidase A (α-Gal A). This disorder belongs to a group of diseases known as lysosomal storage disorders."
Answered by AI
What does Galafold treat?
"The FDA has approved Galafold to treat adults with a certain variant of the galactosidase alpha gene. This is the first oral treatment for Fabry disease that has been approved by the FDA."
Answered by AI