Galafold
Galafold (migalastat) is an oral medication that's used for a rare, inherited condition called Fabry disease. It was approved by the FDA through its Accelerated Approval Program. You'll need to visit a genetic counselor to see if this medication is right for you. Galafold (migalastat) is only available at specialty pharmacies, so your provider or pharmacist will need to help you set that up.
What is Galafold (migalastat)?
What is Galafold (migalastat) used for?
- Treatment of Fabry disease in certain people
How Galafold (migalastat) works
Galafold (migalastat) is a pharmacologic chaperone. It attaches to a protein in your body called alpha-galactosidase A (alpha-Gal A), which doesn’t work as well as it should in Fabry disease. By attaching to alpha-Gal A, Galafold (migalastat) helps the protein travel to where it needs to go to break down extra fatty waste in the body.
Drug facts
Side effects of Galafold (migalastat)
The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.
Common Side Effects
- Headache (35%)
- Stuffy nose, runny nose, or sore throat (18%)
- Urinary tract infection (UTI, 15%)
- Nausea (12%)
- Fever (12%)
Less Common Side Effects
- Belly pain
- Back pain
- Cough
- Diarrhea
- Vomiting
- Nosebleed
The following Galafold (migalastat) side effects have also been reported
Side effects that you should report to your care team as soon as possible:
- Allergic reactions—skin rash, itching, hives, swelling of the face, lips, tongue, or throat
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
- Headache
- Nausea
- Runny or stuffy nose
- Sore throat
- Stomach pain
Pros and cons of Galafold (migalastat)
Pros
- The first and only oral medication for Fabry disease
- Only taken once every other day so it's convenient for people with a busy schedule
- Copay savings program available for people who are eligible
Cons
- Only available as a brand medication, so it might be expensive
- Can't be crushed or chewed, so it might not be a good option for people who can't swallow pills
- Not recommended for use in people with severe kidney disease
Pharmacist tips for Galafold (migalastat)
- Take Galafold (migalastat) on an empty stomach. Don't eat any food at least 2 hours before and 2 hours after this medication. Taking Galafold (migalastat) too close to food can lower the amount of medication that gets absorbed into your body. It's okay to drink clear liquids, such as water, juices without pulp, soda, or tea, during this time when you can't eat.
- Swallow Galafold (migalastat) capsules whole. Don't cut, crush, or chew the capsules. Doing so can affect the way the medication works in your body.
- Take Galafold (migalastat) at the same time every other day to be consistent and to make sure an effective amount of medication is in your body at all times.
- Don't take Galafold (migalastat) two days in a row to prevent too much medication in your body.
- On your first day of using each new blister card, write down the start date to help you remember when to take the next dose. On the days that you don't need to take the medication, press down on the cardboard circle on the top row of the blister card. Removing the cardboard circle can help you remember which day you don't take Galafold (migalastat).
- Keep Galafold (migalastat) capsules in the blister card. Store this medication in a dry and cool place to protect it from moisture.
Common questions about Galafold (migalastat)
Fabry disease is a rare, inherited condition caused by changes in the GLA gene. The GLA gene provides instructions for your body on how to make a protein called alpha-Gal A. Alpha-Gal A helps get rid of certain fats from your body. People with Fabry disease often don't have much alpha-Gal A that function correctly. As a result, the fats start to collect in blood vessels, the kidneys, the heart, and many other organs. Extra fats can cause nerve pain; nausea and vomiting; small, red spots on the skin; and hearing loss. Over time, they can also cause serious problems, such as kidney failure, irregular heartbeat, and stroke. Everyone can have different signs and symptoms of this condition. If you have Fabry disease, it's important to follow up with a provider often, even if you don't have any signs and symptoms.
The FDA's Accelerated Approval Program allows a new medication to be approved faster than usual if it fulfills an unmet need or if it treats serious or life-threatening conditions. These approvals are based on lab measurements that are thought to predict health outcomes before the medication shows clear benefits. For Galafold (migalastat), the medication lowered the amount of extra fats in the kidneys, which can potentially predict better survival chance in people with Fabry disease. After accelerated approval, the manufacturer is required to complete clinical studies to confirm beneficial health outcomes (in this case, if Galafold (migalastat) improved health outcomes in people with Fabry disease). Depending on the results of these studies, the FDA can either give the medication a full approval or pull it off the market. For more information on the FDA Accelerated Approval Program, check out this article.
The time of day you take Galafold (migalastat) doesn’t matter, as long as it’s the same time on the days you need to take it. You also need to take Galafold (migalastat) on an empty stomach, so you can’t eat for at least 2 hours before and after taking the medication. Because of this, you might want to consider picking a time that works best for your eating schedule. For example, if you typically eat more than 2 hours before you go to sleep, then you can take Galafold (migalastat) every other night at bedtime.
If you’ve forgotten to take Galafold (migalastat) and it’s within 12 hours of the usual time you take it, take your dose as soon as possible. If it's been more than 12 hours, skip the missed dose. Take the next dose of Galafold (migalastat) on your next scheduled day and time.
Galafold (migalastat) hasn’t been studied in human pregnancies and breastfeeding mothers. Before starting Galafold (migalastat), talk with your provider to discuss the benefits and risks of taking this medication. Additionally, if you have Fabry disease and are pregnant or are breastfeeding a baby up to 1 year of age, you’re encouraged to enroll in the Fabry Pregnancy Registry. This registry monitors and collects information about your and your baby’s health. Learn more about this registry by calling 1-888-239-0758 or visit this website. Please talk with your provider if you’re interested in participating in the registry.
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Galafold (migalastat) dosage
Typical dosage for Galafold (migalastat)
The typical dose is 1 capsule (123 mg) by mouth every other day on an empty stomach. Don't eat food at least 2 hours before and 2 hours after taking this medication.
