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Overview: Pombiliti is a medicine used with another medicine called Opfolda for certain people with late-onset Pompe disease. If you miss a dose of Opfolda, call your health care provider right away. You may not be able to get your Pombiliti dose. Tell your health care provider right away if you feel dizzy, warm, or itchy when you get a dose or within a few hours after getting this medicine. Get emergency help if you have trouble breathing or swelling of the face, lips, mouth, tongue, or throat.

Uses

What is Pombiliti used for?

Pombiliti (cipaglucosidase alfa-atga) is commonly used with another medicine for certain people with late-onset Pompe disease.

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

How does Pombiliti work (mechanism of action)?

People with Pompe disease have a problem with a specific protein (enzyme) that breaks down glycogen to glucose. Pombiliti is a medicine that provides this enzyme in a form that allows it to enter cells where it normally is found. Provision of this enzyme allows for glycogen to change into glucose. This allows for the build-up of glycogen within cells to stop.

How is Pombiliti supplied (dosage forms)?

Pombiliti is available in an injectable form that is given by a healthcare provider.

How should I store Pombiliti?

Pombiliti is usually given by a health care provider in a hospital or clinic. You will not store it at home.

Side Effects

What are the most common side effects of Pombiliti?

The most common side effects of Pombiliti are listed below. These side effects reflect use with another medicine called Opfolfa. 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 Pombiliti 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 Pombiliti?

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

Severe Allergic Reactions. Pombiliti may cause allergic reactions, which can be serious. 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

Infusion Reactions. Pombiliti can cause reactions that can be serious when it is given to you. Your infusion may need to be slowed or stopped if you have these reactions. Tell your health care provider right away if you have any of the following symptoms when you get your infusion or within a few hours of getting a dose. 

  • Chills
  • Dizziness or lightheadedness
  • Redness of your face and neck
  • Trouble breathing or swallowing
  • Hives or itching
  • Swelling of the throat

Warnings & Precautions

Who should not use Pombiliti?

Allergies to Ingredients. People who are allergic to any of the following should not use Pombiliti.

  • Cipaglucosidase alfa
  • Pombiliti
  • Any of the ingredients in the specific product dispensed

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

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

What should I know about Pombiliti before using it?

Pombiliti is usually given by a health care provider into a vein as an intravenous (IV) infusion in a hospital or clinic. This medicine is used with another medicine called Opfolda. If you miss a dose of Opfolda, call your health care provider right away. You may not be able to get your Pombiliti dose.

Your health care provider may give you certain medicines before your dose of Pombiliti to help reduce the risk of side effects. Take these medicines as your health care provider recommends.

Pombiliti 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.

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

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 Pombiliti is right for you.

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

Current and Past Health Conditions. Tell your health care provider if you have any of the following.

Pregnancy. Pombiliti is used with another medicine called Opfolda, 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 Pombiliti passes into breast milk. Do not breastfeed during your treatment with Pombiliti. Tell your health care provider if you are breastfeeding or plan to breastfeed.

How can I get more information about Pombiliti?

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

Interactions

Does Pombiliti interact with foods or drinks?

There are no known interactions between Pombiliti and foods or drinks.

It is unknown if drinking alcohol will affect Pombiliti.

Does Pombiliti interact with other medicines (drug interactions)?

There are no known interactions between Pombiliti 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 Pombiliti?

If you or someone else has used too much Pombiliti, 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 Pombiliti?

Pombiliti is usually given by a health care provider in a hospital. Your health care provider will make sure you are receiving Pombiliti as scheduled.

If you miss an appointment to get your Pombiliti dose or you forget to take Ofolda at the correct time, call your health care provider right away.