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Overview: Opfolda is a medicine used with another medicine called Pombiliti for certain people with late-onset Pompe disease. Opfolda helps Pombiliti get inside your cells and must be taken as directed. If you miss a dose, call your health care provider right away. You may not be able to get your Pombiliti dose.

Uses

What is Opfolda used for?

Opfolda (miglustat) is commonly used with another medicine for certain people with late-onset Pompe disease. 

Opfolda may also be used for other conditions as determined by your health care provider.

How does Opfolda work (mechanism of action)?

People with Pompe disease have a problem with a specific protein (enzyme) that breaks down glycogen to glucose. Opfolda is given with a medicine called Pombiliti. Opfolda binds and stabilizes Pombiliti in your blood, so more Pombiliti can get into your cells. Once inside the cells, Opfolda and Pombiliti separate. Opfolda helps treat people with late-onset Pompe disease by helping Pombiliti to not get broken down in the blood. 

How is Opfolda supplied (dosage forms)?

Opfolda is available as a 65 mg oral capsule.

How should I store Opfolda?

Opfolda should be stored in the original container to protect it from light at room temperature, between 68 F and 77 F (20 C and 25 C). It can be exposed to temperatures between 59 F and 86 F (15 C and 30 C) for shorter periods of time, such as when transporting it. Store in a cool, dry place.

Side Effects

What are the most common side effects of Opfolda?

The most common side effects of Opfolda are listed below. These side effects reflect use with Pombiliti. Tell your health care provider if you have any of these side effects that bother you.

  • Headache
  • Diarrhea
  • Tiredness
  • Nausea or stomach pain
  • Fever

There may be other side effects of Opfolda that are not listed here. Contact your health care provider if you think you are having a side effect of a medicine. In the U.S., you can report side effects to the FDA at www.fda.gov/medwatch or by calling 800-FDA-1088 (800-332-1088). In Canada, you can report side effects to Health Canada at www.health.gc.ca/medeffect or by calling 866-234-2345.

What are the serious side effects of Opfolda?

While less common, the most serious side effects of Opfolda are described below, along with what to do if they happen.

Severe Allergic Reactions. Opfolda may cause allergic reactions, which can be serious. Stop taking Opfolda and get help right away if you have any of the following symptoms of a serious allergic reaction.

  • Breathing problems or wheezing
  • Racing heart
  • Fever or general ill feeling
  • Swollen lymph nodes
  • Swelling of the face, lips, mouth, tongue, or throat
  • Trouble swallowing or throat tightness
  • Itching or skin rash
  • Bumps on the skin called hives that can be red, pink, white, or brown depending on your skin tone
  • Nausea or vomiting
  • Dizziness, feeling lightheaded, or fainting
  • Stomach cramps
  • Joint pain

Warnings & Precautions

Who should not use Opfolda?

Allergies to Ingredients. People who are allergic to any of the following should not take Opfolda.

  • Miglustat
  • Opfolda
  • Any of the ingredients in the specific product dispensed

Your pharmacist can tell you all of the ingredients in Opfolda.

Pregnancy. Do not use Opfolda if you are pregnant. If you become pregnant, stop using Opfolda and contact your healthcare provider right away. See the Pregnancy section below for more details.

What should I know about Opfolda before using it?

Do not take Opfolda unless it has been prescribed to you by a health care provider. Take it as prescribed.

Do not share Opfolda with other people, even if they have the same condition as you. It may harm them.

Keep Opfolda out of the reach of children.

Opfolda may make it hard for you to get pregnant or get your partner pregnant. If you may want to have children one day, talk to your health care provider.

This medicine is not used on a regular basis. Consider using an app or alarm to help you remember when to take it.

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

What should I tell my health care provider before using Opfolda?

Tell your health care provider about all of your health conditions and any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using. This will help them know if Opfolda is right for you.

In particular, make sure that you discuss any of the following.

Kidney Problems. Tell your health care provider if you have any kidney problems. Your health care provider may need to check how well your kidneys are working. Keep all appointments to have your blood checked.

Pregnancy. Opfolda is used with another medicine called Pombiliti, and the treatment may harm an unborn baby. A negative pregnancy test is necessary before starting treatment. If you can become pregnant, use effective birth control during your treatment and for at least 60 days after your last dose. Tell your health care provider right away if you are pregnant, think you may be pregnant, or plan to become pregnant. 

Breastfeeding. It is not known if Opfolda passes into breast milk. Do not breastfeed during your treatment with Opfolda. Tell your health care provider if you are breastfeeding or plan to breastfeed.

How can I get more information about Opfolda?

For more information about Opfolda, you can visit the drugmaker’s website at www.pombilitiopfolda.com or call them at 877-426-4287.

Interactions

Does Opfolda interact with foods or drinks?

Take Opfolda on an empty stomach. This means do not drink or eat anything 2 hours before and 2 hours after taking this medicine. Swallow Opdolda with unsweetened drinks, such as water, tea, or coffee without cream, sugar, or sweeteners.

It is unknown if drinking alcohol will affect Opfolda.

Does Opfolda interact with other medicines (drug interactions)?

There are no known interactions between Opfolda and other medicines. Always tell your health care provider about any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using.

Does interact with other drugs you are taking?

Enter your medication into the WebMD interaction checker

Overdose/Missed Dose

What should I do if I accidentally use too much Opfolda?

If you or someone else has used too much Opfolda, get medical help right away, call 911, or contact a Poison Control center at 800-222-1222.

What should I do if I miss a dose of Opfolda?

If you miss a dose of Opfolda, call your health care provider right away. This medicine is used with another medicine called Pombiliti. You may not be able to get your Pombiliti dose if you miss your Opfolda dose.