Galafold (Migalastat) belongs to the class of medications called alimentary tract and metabolism products.

It is used for long-term treatment of Fabry disease. Fabry disease is a condition where changes to certain genes prevent the body from producing enough of the enzyme alpha-galactosidase A. This enzyme helps to break down a particular fatty substance (globotriaosylceramide – GL-3) that builds up in the kidneys, heart, and other organs in Fabry disease.

Galafold (Migalastat) works by preventing alpha-galactosidase A from being broken down in the body, leaving more enzyme available for the body to use. It also fixes a defect in the enzyme, allowing it to work better. Both of these actions contribute to reducing the build-up of GL-3 in the body.

Galafold (Migalastat) may be available under multiple brand names and/or in several different forms. Any specific brand name of Galafold (Migalastat) may not be available in all of the forms or approved for all of the conditions discussed here. As well, some forms of Galafold (Migalastat) may not be used for all of the conditions discussed here.

Your doctor may have suggested Galafold (Migalastat) for conditions other than those listed in these drug information articles. If you have not discussed this with your doctor or are not sure why you are taking Galafold (Migalastat), speak to your doctor. Do not stop taking Galafold (Migalastat) without consulting your doctor.

Do not give Galafold (Migalastat) to anyone else, even if they have the same symptoms as you do. It can be harmful for people to take Galafold (Migalastat) if their doctor has not prescribed it.

The usual adult dose of Galafold (Migalastat) is one 123 mg capsule taken by mouth, every other day, at approximately the same time each day. To get the best effect from Galafold (Migalastat), it must be taken on an empty stomach. Do not eat or drink anything other than clear liquids for 2 hours before or 2 hours after taking Galafold (Migalastat).

Swallow the capsules whole. Do not crush, chew or cut the capsule.

Many things can affect the dose of medication that a person needs, such as body weight, other medical conditions, and other medications. If your doctor has recommended a dose different from the ones listed here, do not change the way that you are taking the medication without consulting your doctor.

It is important to take Galafold (Migalastat) exactly as prescribed by your doctor. Taking more of Galafold (Migalastat) than prescribed or taking it more often than prescribed causes it to become less effective.

If you miss a dose, and it is within 12 hours of the time you usually take the medication, take it as soon as possible and continue with your regular schedule. If it has been more than 12 hours since your missed dose, almost time for your next dose, skip the missed dose and continue with your regular dosing schedule. Do not take a double dose to make up for a missed one. If you are not sure what to do after missing a dose, contact your doctor or pharmacist for advice.

Store Galafold (Migalastat) at room temperature, protect it from light and moisture, and keep it out of the reach of children.

Do not dispose of medications in wastewater (e.g. down the sink or in the toilet) or in household garbage. Ask your pharmacist how to dispose of medications that are no longer needed or have expired.

Many medications can cause side effects. A side effect is an unwanted response to a medication when it is taken in normal doses. Side effects can be mild or severe, temporary or permanent.

The side effects listed below are not experienced by everyone who takes Galafold (Migalastat). If you are concerned about side effects, discuss the risks and benefits of Galafold (Migalastat) with your doctor.

The following side effects have been reported by at least 1% of people taking Galafold (Migalastat). Many of these side effects can be managed, and some may go away on their own over time.

Contact your doctor if you experience these side effects and they are severe or bothersome. Your pharmacist may be able to advise you on managing side effects.

• abdominal pain
• appetite changes
• arm, leg, back, or neck pain
• balance problems, unsteadiness
• changed patches of skin pigmentation
• changed sense of touch
• chest discomfort
• Diarrhea
• dizziness
• Dry Mouth
• feeling hot
• flu-like symptoms (e.g., body aches, fatigue, Fever)
• frequent urination
• hair loss
• headache
• heartburn
• itchy eyes
• memory problems
• migraine
• muscle pain, spasms
• muscular chest pain
• nausea
• night sweats
• nosebleeds
• pins and needles in hands or feet
• psoriasis (red, itchy, scaly patches of skin)
• runny nose
• skin rash or redness
• sneezing
• spinning sensation
• sweating
• tiredness
• trouble sleeping
• vision changes
• vomiting
• weight changes

Although most of the side effects listed below don't happen very often, they could lead to serious problems if you do not seek medical attention.

Check with your doctor as soon as possible if any of the following side effects occur:

• aggressiveness, feeling impatient
• blood in the stools
• breathing problems
• enlarged Thyroid gland (swelling at the front of the neck)
• fast, pounding heartbeat
• high blood pressure
• irritable bowel syndrome (e.g., stomach pain or cramping, bloating, diarrhea or Constipation, excessive gas)
• lack of muscle coordination
• low blood sugar (e.g., cold sweat, cool pale skin, headache, fast heartbeat, weakness)
• movement disorders (e.g., unintended muscle movement, decreased coordination, tremor)
• puffiness in arms, legs, ankles, weight gain
• signs of Depression (e.g., poor concentration, changes in weight, changes in sleep, decreased interest in activities, thoughts of suicide)
• signs of liver problems (e.g., nausea, vomiting, diarrhea, loss of appetite, weight loss, yellowing of the skin or whites of the eyes, dark urine, pale stools)
• swelling feet and legs
• symptoms of gallstones (e.g., intermittent, severe, dull pain in the upper right part of the abdomen; nausea; vomiting; intolerance of fatty or greasy foods)
• symptoms of a urinary tract infection (e.g. pain when urinating, urinating more often than usual, low back or flank pain)
• urine cloudiness, foaming urine

Stop taking the medication and seek immediate medical attention if any of the following occur:

• irregular heartbeat (e.g., fast, pounding heartbeat, fainting, lightheadedness, shortness of breath)
• lack of oxygen to heart muscle, signs of Heart Attack (e.g., sudden chest pain or pain radiating to back, down arm, jaw; sensation of fullness of the chest; nausea; vomiting; sweating; anxiety)

Some people may experience side effects other than those listed. Check with your doctor if you notice any symptom that worries you while you are taking Galafold (Migalastat).

Each size 2 hard capsule with an opaque blue cap and opaque white body, with "A1001" printed in black, contains 123 mg of Galafold (Migalastat), equivalent to 150 mg of Galafold (Migalastat) hydrochloride. Nonmedicinal ingredients: black printing ink, gelatin, indigotine (FD&C Blue No. 2), magnesium stearate, pregelatinized maize starch, and titanium dioxide.

Do not take Galafold (Migalastat) if you are allergic to Galafold (Migalastat) or any ingredients of the medication.

There may be an interaction between Galafold (Migalastat) and any of the following:

• agalsidase

If you are taking any of these medications, speak with your doctor or pharmacist. Depending on your specific circumstances, your doctor may want you to:

• stop taking one of the medications,
• change one of the medications to another,
• change how you are taking one or both of the medications, or
• leave everything as is.

An interaction between two medications does not always mean that you must stop taking one of them. Speak to your doctor about how any drug interactions are being managed or should be managed.

Medications other than those listed above may interact with Galafold (Migalastat). Tell your doctor or prescriber about all prescription, over-the-counter (non-prescription), and herbal medications you are taking. Also tell them about any supplements you take. Since caffeine, alcohol, the Nicotine from cigarettes, or street drugs can affect the action of many medications, you should let your prescriber know if you use them.

Before you begin using a medication, be sure to inform your doctor of any medical conditions or Allergies you may have, any medications you are taking, whether you are pregnant or breast-feeding, and any other significant facts about your health. These factors may affect how you should use Galafold (Migalastat).

Enzyme replacement therapy: Galafold (Migalastat) should not be used by people receiving agalsidase, an enzyme replacement therapy for treating Fabry disease. This combination causes an increase in the amount of agalsidase to build up in the body, causing side effects and reduced effectiveness of the Galafold (Migalastat) and possibly worsening of the disease.

Kidney function: Kidney Disease or reduced kidney function may cause Galafold (Migalastat) to build up in the body. If Galafold (Migalastat) builds up in the body, it becomes less effective, as well as causing increased side effects. If you have reduced kidney function or kidney disease, discuss with your doctor how Galafold (Migalastat) may affect your medical condition, how your medical condition may affect the dosing and effectiveness of Galafold (Migalastat), and whether any special monitoring is needed.

Pregnancy: Galafold (Migalastat) should not be used during pregnancy unless the benefits outweigh the risks. If you become pregnant while taking Galafold (Migalastat), contact your doctor immediately.

Breast-feeding: It is not known if Galafold (Migalastat) passes into breast milk. If you are a breast-feeding mother and are taking Galafold (Migalastat), it may affect your baby. Talk to your doctor about whether you should continue breast-feeding.

Children: The safety and effectiveness of using Galafold (Migalastat) have not been established for children.