Migalastat

Migalastat is a prescription medicine used for the treatment of Fabry disease. 

• Migalastat is available under the following different brand names: Galafold

Common side effects of Migalastat include:

• headache,
• nausea,
• fever, and
• cold symptoms (stuffy nose, sneezing, sore throat)

Serious side effects of Migalastat include:

• hives,
• difficulty breathing,
• swelling of the face, lips, tongue, or throat, and
• pain or burning while urinating
• seizure, and
• loss of consciousness

Rare side effects of Migalastat include:

• none

Seek medical care or call 911 at once if you have the following serious side effects:

• Severe headache, confusion, slurred speech, arm or leg weakness, trouble walking, loss of coordination, feeling unsteady, very stiff muscles, high fever, profuse sweating, or tremors;
• Serious eye symptoms such as sudden vision loss, blurred vision, tunnel vision, eye pain or swelling, or seeing halos around lights;
• Serious heart symptoms include fast, irregular, or pounding heartbeats; fluttering in the chest; shortness of breath; sudden dizziness, lightheadedness, or passing out. 

This is not a complete list of side effects and other serious side effects or health problems that may occur because of the use of this drug. Call your doctor for medical advice about serious side effects or adverse reactions. You may report side effects or health problems to FDA at 1-800-FDA-1088.

Adult dosage

Capsule

• 123 mg

Fabry Disease

Adult dosage

• 123 mg orally once every other day at the same time of day

Dosage Considerations – Should be Given as Follows: 

• See “Dosages”

If your medical doctor is using this medicine to treat your pain, your doctor or pharmacist may already be aware of any possible drug interactions and may be monitoring you for them. Do not start, stop, or change the dosage of any medicine before checking with your doctor, health care provider, or pharmacist first.

• Migalastat has severe interactions with no other drugs.
• Migalastat has serious interactions with no other drugs.
• Migalastat has moderate interactions with no other drugs.
• Migalastat has minor interactions with no other drugs.

This information does not contain all possible interactions or adverse effects. Visit the RxList Drug Interaction Checker for any drug interactions. Therefore, before using this product, tell your doctor or pharmacist about all your products. Keep a list of all your medications with you and share this information with your doctor and pharmacist. Check with your healthcare professional or doctor for additional medical advice, or if you have health questions or concerns.

Contraindications

• None reported by the manufacturer

Effects of drug abuse

• None

Short-Term Effects

• See “What Are Side Effects Associated with Using Migalastat?”

Long-Term Effects

• See “What Are Side Effects Associated with Using Migalastat?”

Cautions

• This product was approved under accelerated approval based on a reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Pregnancy and Lactation

• Current study collects data on pregnant women with Fabry disease, either exposed or unexposed to GALAFOLD; healthcare providers are encouraged to register patients or obtain additional information by contacting the Pregnancy Coordinating Center at 1-888-239-0758; email fabr
• Available data are not sufficient to assess drug-associated risks of major birth defects, miscarriage, or adverse maternal or fetal outcomes; in animal reproduction studies, no adverse developmental effects were observed
• Infertility
• Effects on fertility in humans not studied; transient and fully reversible infertility in male rats associated with treatment at systemic exposure (AUC) equivalent to human exposure at recommended dose; complete reversibility was seen at 4 weeks after termination of treatment; drug did not affect fertility in female rats
• Lactation
  • Current study collects data on the effects of GALAFOLD on lactation for women with Fabry disease and their neonates and infants up to 1 year of age who are exposed through breast milk; healthcare providers are encouraged to register patients or obtain additional information by contacting Pregnancy Coordinating Center at 1-888-239-0758, email [email protected], or visit www.fabrypregnancyregistry.com
  • There are no human data available on the presence of the drug in human milk, the effects on the breastfed infant, or on milk production; the drug is present in the milk of lactating rats; when a drug is present in animal milk, it is likely that the drug will be present in human milk; developmental and health benefits of breastfeeding should be considered along with mother’s clinical need for therapy and any potential adverse effects on the breastfed child from the drug or from an underlying maternal condition.