Galafold is a brand-name prescription medication. It’s approved by the Food and Drug Administration (FDA) to treat Fabry disease in certain adults. They should have a specific change in a gene called galactosidase alpha (GLA). For details, see the “Galafold for Fabry disease” section below.

Fabry disease is a rare, inherited condition that causes fat to build up throughout your body. The fat can gather in areas such as the kidneys, nervous system, and cardiovascular system. (“Cardiovascular” means related to the heart and blood vessels.)

Drug details

The active drug in Galafold is migalastat, which is in a medication class called alpha-galactosidase A (alpha-Gal A) pharmacological chaperones.

Galafold comes as a capsule that you swallow. The drug is available in one strength: 123 milligrams (mg).

FDA approval

In 2018, the FDA granted accelerated approval to Galafold as the first oral treatment for Fabry disease.

Accelerated approval is based on information from early clinical trials of a drug. The FDA will decide whether to grant full approval of Galafold after additional clinical trials have been completed.

Typically, drugs receive approval from the FDA after extensive studies have been completed. But for some drugs, including Galafold, a drug’s approval is granted before all of the studies have been done.

Accelerated approval is granted for certain drugs that are used to treat conditions that don’t have a lot of successful treatment options. For example, with Fabry disease, other treatment options involve medications given as an intravenous (IV) infusion. This is an injection into a vein that’s given over a period of time.

Effectiveness

For information about the effectiveness of Galafold, see the “Galafold for Fabry disease” section below.

Galafold is available only as a brand-name medication. It’s not currently available in generic form.

A generic drug is an exact copy of the active drug in a brand-name medication. Generics usually cost less than brand-name drugs.

As with all medications, the cost of Galafold can vary. To find current prices for Galafold in your area, check out WellRx.com.

The cost you find on WellRx.com is what you may pay without insurance. The actual price you’ll pay depends on your insurance plan, your location, and the pharmacy you use.

It’s important to note that you’ll likely have to get Galafold at a specialty pharmacy. This type of pharmacy is authorized to carry specialty medications. These are drugs that may be expensive or may require help from healthcare professionals to be used safely and effectively.

Before approving coverage for Galafold, your insurance company may require you to get prior authorization. This means that your doctor and insurance company will need to communicate about your prescription before the insurance company will cover the drug. The insurance company will review the prior authorization request and decide if the drug will be covered.

If you’re not sure if you’ll need to get prior authorization for Galafold, contact your insurance company.

Financial and insurance assistance

If you need financial support to pay for Galafold, or if you need help understanding your insurance coverage, help is available.

Amicus Therapeutics, the manufacturer of Galafold, offers a program called Amicus Assist, which provides cost assistance options. For more information and to find out if you’re eligible for support, call 833-AMICUS-A (833-264-2872) or visit the program website.

Generic version

Galafold is not available in a generic form. A generic drug is an exact copy of the active drug in a brand-name medication. Generics tend to cost less than brand-name drugs.

The Food and Drug Administration (FDA) approves prescription drugs such as Galafold to treat certain conditions.

Galafold is approved to treat Fabry disease in adults who have a certain variant (change) in a gene called galactosidase alpha gene (GLA). This variant allows the gene to be treated with Galafold. The drug is the first FDA-approved oral treatment for Fabry disease.

Fabry disease explained

Fabry disease is inherited. The change in the GLA gene causes a deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A). An enzyme is a protein that aids chemical changes in your body. And the deficiency means that you have too little of the enzyme.

Without enough of the enzyme, your body has problems breaking down lipids (fats). This causes fat to build up throughout your body. The fat can gather in areas such as your kidneys, nervous system, and cardiovascular system. (“Cardiovascular” means related to the heart and blood vessels.)

One of the fats that builds up in Fabry disease is called globotriaosylceramide (GL-3). Galafold works to decrease the number of GL-3 buildups as measured by a kidney biopsy (removal of tissue for examination). It’s thought that reducing the buildup could help ease symptoms of Fabry disease.

For more information on how Galafold works, see the “How Galafold works” section below.

Symptoms of Fabry disease

Fabry disease mainly affects males.* Females* usually have no symptoms or the symptoms are much milder than in males.

Symptoms of Fabry disease in males can include:

  • foot and hand pain
  • rash that appears as red or dark spots
  • clouding of the cornea (the clear part of the front of the eye)
  • sweating less than usual when hot

* Sex and gender exist on spectrums. Use of the terms “male” and “female” in this article refers to sex assigned at birth.

Effectiveness for Fabry disease

In clinical studies of people with Fabry disease, researchers found Galafold to be effective at reducing the number of GL-3 buildups. This was according to kidney biopsies. The people in the studies had a certain variant in their GLA gene that makes the gene able to be treated with Galafold.

It’s important to note that a relationship between reducing GL-3 lipid buildups and relief of Fabry disease symptoms hasn’t been proven. Galafold is approved to treat Fabry disease based on the results of early clinical trials.* Researchers saw that the drug is effective in lowering the number of GL-3 buildups, which may help relieve symptoms of Fabry disease. But the researchers didn’t look at whether the drug actually eases Fabry disease symptoms.

* For more information, see “FDA approval” in the “About Galafold” section above.

Galafold and children

Galafold is only approved for use in adults. The drug hasn’t been studied in children, so it is not known if Galafold is safe or effective for them.

If you have questions about Fabry disease treatment options for your child, talk with their doctor.

The following information describes dosages that are commonly used or recommended. However, be sure to take the dosage your doctor prescribes for you. Your doctor will determine the best dosage to fit your needs.

Drug forms and strengths

Galafold comes as a capsule that you swallow. It’s available in one strength: 123 milligrams (mg).

Dosage for Fabry disease

The recommended Galafold dosage for Fabry disease is 123 mg taken once every other day. When you take a dose, be sure to take it at the same time of day.

What if I miss a dose?

If you miss a dose and it’s within 12 hours of the regularly scheduled dose time, take the dose as soon as you remember.

If you miss a dose and it’s been more than 12 hours since the regularly scheduled time, skip the missed dose. Then take your next scheduled Galafold dose according to your every-other-day schedule.

You should not change your dosing schedule. Also, you should not “double up” and take more than one capsule to make up for the missed dose. This could increase your risk for side effects. (For information on side effects, see the “Galafold side effects” section below.)

To help make sure that you don’t miss a dose, try using a medication reminder. This can include setting an alarm or timer on your phone or downloading a reminder app. A kitchen timer can work, too.

Will I need to use this drug long term?

Galafold is meant to be used as a long-term treatment. If you and your doctor determine that it is safe and effective for you, you’ll likely take it long term.

Galafold can cause mild or serious side effects. The following lists contain some of the key side effects that may occur while taking this drug. These lists do not include all possible side effects.

For more information about the possible side effects of Galafold, talk with your doctor or pharmacist. They can give you tips on how to deal with any side effects that may be concerning or bothersome.

Note: The Food and Drug Administration (FDA) tracks side effects of drugs it has approved. If you would like to notify the FDA about a side effect you’ve had with Galafold, you can do so through MedWatch.

Mild side effects

Mild side effects* of Galafold can include:

Most of these side effects may go away within a few days or a couple of weeks. But if they become more severe or don’t go away, talk with your doctor or pharmacist.

* This is a partial list of mild side effects from Galafold. To learn about other mild side effects, talk with your doctor or pharmacist, or view Galafold’s patient brochure.
† For more information on this side effect, see “Side effect details” below.

Serious side effects

Serious side effects from Galafold aren’t common, but they can occur. Call your doctor right away if you have serious side effects. Call 911 or your local emergency number if your symptoms feel life threatening or if you think you’re having a medical emergency.

Serious side effects* can include:

* For more information about these side effects, see “Side effect details” below.

Side effect details

Here are some details on certain side effects this drug may cause.

Headache

Headache was the most common side effect in clinical studies of Galafold. To find out how often this side effect occurred in the studies, see the drug’s prescribing information.

Headaches with Galafold are usually mild.

If you have headaches while taking Galafold, talk with your doctor. They may recommend treatments for this side effect, such as cold packs or over-the-counter (OTC) pain relievers. OTC pain relievers may include acetaminophen (Tylenol) or ibuprofen (Advil, Motrin). If your headaches are bothersome or severe, your doctor may have you stop taking Galafold and switch to a different treatment.

Common cold

Symptoms of the common cold are a possible side effect of Galafold. The symptoms may include a runny nose, stuffy nose, and sore throat. This side effect is usually mild.

Symptoms of colds were common in clinical studies. To find out how often this side effect occurred in the studies, see the drug’s prescribing information.

If you have symptoms of the common cold while taking Galafold, talk with your doctor. They may recommend treatments for this side effect, such as OTC medications. The OTC medications may include acetaminophen and ibuprofen. If your symptoms are bothersome or severe, your doctor may recommend you stop taking Galafold and have you switch to a different treatment for Fabry disease.

Urinary tract infection

Urinary tract infection (UTI) is a possible side effect from taking Galafold. UTIs are typically mild but can be severe in rare cases.

Sometimes mild UTIs go away on their own and don’t cause symptoms. But when mild UTIs do cause symptoms, the symptoms can include:

Symptoms of a severe UTI, such as kidney infection or cystitis, can include:

UTIs were common in clinical studies. To find out how often UTIs occurred in the studies, see the drug’s prescribing information.

Talking with your doctor

If you develop symptoms of a mild or severe UTI while taking Galafold, talk with your doctor right away. They’ll typically want to have your urine tested for a UTI. If you do have a UTI, your doctor may prescribe a treatment, such as an antibiotic.

It’s likely that you’ll be able to keep taking Galafold during and after your UTI treatment. But if you have frequent UTIs or a severe UTI, talk with your doctor. They can advise you on whether a different drug might be a better choice for you.

Allergic reaction

Allergic reaction wasn’t reported in clinical studies of Galafold, but some people can have an allergic reaction after taking the drug.

Symptoms of a mild allergic reaction can include:

  • skin rash
  • itchiness
  • flushing (temporary warmth, redness, or deepening of skin color)

A more severe allergic reaction is rare but possible. Symptoms of a severe allergic reaction can include:

  • swelling under your skin, typically in your eyelids, lips, hands, or feet
  • swelling of your tongue, mouth, or throat
  • trouble breathing

Call your doctor right away if you have an allergic reaction to Galafold, as the reaction could become severe. Call 911 or your local emergency number if your symptoms feel life threatening or if you think you’re having a medical emergency.

Galafold is approved to treat Fabry disease in adults who have a certain variant (change) in a gene called galactosidase alpha gene (GLA). This variant allows the gene to be treated with Galafold. The drug is the first FDA-approved oral treatment for Fabry disease.

You may need to see a genetic counselor to confirm your GLA gene has the variant that Galafold treats.

About Fabry Disease

Fabry disease is inherited. The change in the GLA gene causes a deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A). An enzyme is a protein that aids chemical changes in your body. And the deficiency means that you have too little of the enzyme.

Without enough of the enzyme, your body has problems breaking down lipids (fats). This causes fat to build up throughout your body. The fat can gather in areas such as your kidneys, nervous system, and cardiovascular system. (“Cardiovascular” means related to the heart and blood vessels.)

What Galafold does

One of the fats that builds up in Fabry disease is called globotriaosylceramide (GL-3). Galafold works to decrease the number of GL-3 buildups as measured by a kidney biopsy (removal of tissue for examination). It’s thought that reducing the buildup could help ease symptoms of Fabry disease.

It’s important to note that a relationship between reducing GL-3 lipid buildups and the easing of Fabry disease symptoms hasn’t been proven. Galafold is approved to treat Fabry disease based on the results of early clinical trials.* Researchers saw that the drug is effective in lowering the number of GL-3 buildups, which may help relieve symptoms of Fabry disease. But the researchers didn’t look at whether the drug actually eases Fabry disease symptoms.

* For more information, see “FDA approval” in the “About Galafold” section above.

How long does it take to work?

Galafold begins working as soon as you take your first dose. In clinical studies, doctors didn’t check GL-3 buildup with a kidney biopsy until 6 months after people started taking the drug. This is because it can take some time before your body starts producing more alpha-Gal A.

Because Galafold was approved based on early studies, it isn’t yet known if the drug works to relieve symptoms of Fabry disease. So it isn’t known how soon, or whether, you’ll see your symptoms ease.

There isn’t a known interaction between drinking alcohol and taking Galafold.

However, in some adults, drinking alcohol may cause Fabry disease symptoms such as pain. For this reason, you should talk with your doctor. Ask them how much alcohol, if any, is safe for you to drink during your Galafold treatment.

Galafold isn’t currently known to interact with other medications, herbs, supplements, or foods.

But before taking Galafold, you should still talk with your doctor and pharmacist about all prescription, over-the-counter, and other drugs you take. Also tell them about any vitamins, herbs, and supplements you use. Sharing this information can help you avoid potential interactions, including interactions that may be discovered while you’re taking Galafold.

If you have questions about drug interactions that may affect you, ask your doctor or pharmacist.

You should take Galafold according to instructions your doctor or other healthcare professional gives you.

Galafold comes as a capsule that you swallow.

When to take

You’ll likely take Galafold once every other day. You should not take Galafold capsules 2 days in a row.

When you take a dose, be sure to take it at the same time of day.

To help make sure that you don’t miss a dose, try using a medication reminder. This can include setting an alarm or timer on your phone or downloading a reminder app. A kitchen timer can work, too.

The manufacturer of Galafold offers an app, called Galafold MyDay, to help you remember when to take your Galafold dose. You can learn more about the app here.

Taking Galafold with food

You should take Galafold on an empty stomach. You should not eat within 2 hours before or 2 hours after taking a dose. However, you can drink clear liquids during this time. This includes water as well as tea and coffee without cream, sugar, or milk.

Can Galafold be crushed, opened, or chewed?

No, you shouldn’t crush, open, or chew Galafold capsules. You should swallow them whole.

Talk with your doctor or pharmacist if you have difficulty swallowing Galafold capsules. They may recommend ways to make it easier to take your medication. Your doctor may also suggest a different treatment for Fabry disease.

It is not known if Galafold is safe to take while pregnant. This is because there isn’t enough information on the drug’s use during pregnancy. In animal studies, the drug didn’t harm pregnant animals or their offspring. But animal studies don’t always predict how the drug will affect people.

Pregnancy registry

A pregnancy registry is available for females* with Fabry disease who use Galafold while pregnant. This registry collects information on the safety of using Galafold during pregnancy. If you’d like more information on the registry, talk with your doctor. You can also call 888-239-0758 or visit the registry website.

* Sex and gender exist on spectrums. Use of the term “female” in this article refers to sex assigned at birth.

Talking with your doctor

If you’re pregnant or are planning on becoming pregnant, talk with your doctor before taking Galafold. They can review the benefits and risks of the medication with you.

It’s not known if Galafold is safe to take during pregnancy. If you’re sexually active and you or your partner can become pregnant, talk with your doctor about your birth control needs while you’re using Galafold.

For more information about taking Galafold during pregnancy, see the “Galafold and pregnancy” section above.

It’s not known if Galafold is safe to take while breastfeeding. This is because it is not known whether the drug appears in human breast milk. Animal studies have shown that Galafold is present in animal breast milk, but animal studies don’t always predict what will happen in people.

Pregnancy registry with breastfeeding information

There is a pregnancy registry on females* with Fabry disease, including those who use Galafold while breastfeeding. This registry helps collect information on the safety of using Galafold while breastfeeding. If you’d like more information on the registry, including how to join, you can call 888-239-0758 or visit the registry website.

* Sex and gender exist on spectrums. Use of the term “female” in this article refers to sex assigned at birth.

Talking with your doctor

If you’re breastfeeding or interested in breastfeeding, talk with your doctor before taking Galafold. They can review the benefits and risks of the medication with you.

Here are answers to some frequently asked questions about Galafold.

Does Galafold cure Fabry disease?

No, Galafold isn’t a cure for Fabry disease. There currently is no cure for Fabry disease.

Galafold is approved to treat Fabry disease based on the results of early clinical trials.* Researchers saw that the drug is effective in reducing the buildup of a specific type of fat, which may help relieve symptoms of Fabry disease. But the researchers didn’t look at whether the drug actually eases Fabry disease symptoms.

For more information on how Galafold works, see the “How Galafold works” section above. You may also talk with your doctor or pharmacist.

* For more information, see “FDA approval” in the “About Galafold” section above.

Why should I see a genetic counselor before starting treatment with Galafold?

Before you use Galafold, a genetic counselor will look at a gene called galactosidase alpha gene (GLA). They need to confirm that the gene has a variant (change). This variant allows the gene to be able to be treated with Galafold. If you don’t have this variant, you won’t be able to take Galafold for Fabry disease.

For more information on seeing a genetic counselor, speak with your doctor.

Why do I have to fast to take my doses of Galafold?

You need to fast to take your doses of Galafold because food can prevent your body from absorbing all of your Galafold dose. This can cause Galafold to not work. In clinical studies, people took the medication while fasting.

If you have difficulty fasting for your Galafold dose, talk with your doctor. They may recommend exploring other treatment options for Fabry disease.

Before taking Galafold, talk with your doctor about your health history. Galafold may not be right for you if you have certain medical conditions or other factors affecting your health. These include:

Severe kidney problems and dialysis. Your body gets rid of Galafold through your kidneys. If you have severe kidney problems, whether or not you use dialysis, you should not take Galafold. This is because the drug hasn’t been studied in people with severe kidney problems. It isn’t known if having severe kidney problems could cause the level of Galafold to become too high in your body. A high level could increase the risks of side effects.

If you have mild or moderate kidney problems, you should be able to take Galafold. Before you begin treatment with Galafold, talk with your doctor about any kidney problems you have or had.

Allergic reaction. If you’ve had an allergic reaction to Galafold or any of its ingredients, you should not take Galafold. Ask your doctor about other medications that might be better options for you.

Pregnancy. It’s not known whether Galafold is safe to take while pregnant. For more information, see the “Galafold and pregnancy” section above.

Breastfeeding. It’s not known whether Galafold is safe to take while breastfeeding. For more information, see the “Galafold and breastfeeding” section above.

Note: For more information about the potential negative effects of Galafold, see the “Galafold side effects” section above.

Do not use more Galafold than your doctor recommends. For some drugs, doing so may lead to unwanted side effects or overdose.

What to do in case you take too much Galafold

If you think you’ve taken too much of this drug, call your doctor. You can also call the American Association of Poison Control Centers at 800-222-1222 or use their online tool. But if your symptoms are severe, call 911 or your local emergency number, or go to the nearest emergency room right away.

When you get Galafold from the pharmacy, the pharmacist will add an expiration date to the label on the packaging. This date is typically 1 year from the date they dispensed the medication.

The expiration date helps guarantee that the medication is effective during this time. The current stance of the Food and Drug Administration (FDA) is to avoid using expired medications. If you have unused medication that has gone past the expiration date, talk with your pharmacist about whether you might still be able to use it.

Storage

How long a medication remains good to use can depend on many factors, including how and where you store the medication.

You should store Galafold tablets at room temperature from 68°F to 77°F (20°C to 25°C), in the original packaging away from light. For short periods of time, such as while traveling, you may store Galafold capsules between 59°F and 86°F (15°C and 30°C). Be sure to keep the medication in its original packaging and return it to room temperature as soon as possible.

Avoid storing this medication in areas where it could get damp or wet, such as bathrooms.

Disposal

If you no longer need to take Galafold and have leftover medication, it’s important to dispose of it safely. This helps prevent others, including children and pets, from taking the drug by accident. It also helps keep the drug from harming the environment.

This article provides several useful tips on medication disposal. You can also ask your pharmacist for information about how to dispose of your medication.

Disclaimer: Medical News Today has made every effort to make certain that all information is factually correct, comprehensive, and up to date. However, this article should not be used as a substitute for the knowledge and expertise of a licensed healthcare professional. You should always consult your doctor or other healthcare professional before taking any medication. The drug information contained herein is subject to change and is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects. The absence of warnings or other information for a given drug does not indicate that the drug or drug combination is safe, effective, or appropriate for all patients or all specific uses.