Pombiliti
Pombiliti (cipaglucosidase alfa) is an enzyme replacement therapy that treats Pompe disease in adults. It's given as an infusion once every 2 weeks by a healthcare provider. You need to take Opfolda (miglustat) an hour before each infusion; this is an oral medication that helps Pombiliti (cipaglucosidase alfa) work better. Some common side effects of this combination treatment include headache and diarrhea. The Pombiliti (cipaglucosidase alfa) infusion itself can put you at risk throat swelling, hives, and other serious infusion-related reactions.
What is Pombiliti (cipaglucosidase alfa)?
What is Pombiliti (cipaglucosidase alfa) used for?
- Late-onset Pompe disease in adults weighing at least 88 lbs (40 kg) - in combination with Opfolda (miglustat)
How Pombiliti (cipaglucosidase alfa) works
People who have Pompe disease don't have enough of an enzyme (protein) called acid alpha-glucosidase (GAA). This enzyme typically helps convert glycogen stored in cells into sugars to be used for energy. Without enough GAA, glycogen builds up in the cells and can cause problems, such as muscle weakness.
Pombiliti (cipaglucosidase alfa-atga) is an enzyme replacement therapy (ERT) used to replace GAA in people with Pompe disease. It's given along with an enzyme stabilizer called Opfolda (miglustat) that prevents Pombiliti from breaking down too quickly in the body.
Drug facts
Common questions about Pombiliti (cipaglucosidase alfa)
Opfolda (miglustat) is an enzyme stabilizer. It prevents Pombiliti (cipaglucosidase alfa), an enzyme, from getting broken down too quickly in the body. Opfolda (miglustat) is typically taken 1 hour before Pombiliti (cipaglucosidase alfa). If you miss your dose of Opfolda (miglustat), your provider might ask you to reschedule your Pombiliti (cipaglucosidase alfa) infusion.
Pombiliti (cipaglucosidase alfa) is given as a slow infusion through the veins by a healthcare provider at their office or at an infusion center.
The typical length of the Pombiliti (cipaglucosidase alfa) infusion is about 4 hours. But it could take longer if you experience infusion-related reactions (e.g., hives, chills, or low blood pressure) because your provider might need to stop or slow the infusion. They might also give you medications like acetaminophen (Tylenol), diphenhydramine (Benadryl), or a steroid, to help relieve these infusion-related symptoms. Let your care team know right away if you experience any reactions during the infusion.
Since Pompe disease is a genetic condition, you'll need to receive treatment for your entire life. Your provider will likely ask you to continue Pombiliti (cipaglucosidase alfa) as long as it's helping your condition and you're not having severe side effects. If you have questions about treatment options for Pompe disease, talk with your provider.
No, Pombiliti (cipaglucosidase alfa) and Opfolda (miglustat) must be avoided during pregnancy. Studies suggest this combination can cause severe harm to babies if taken by mothers during pregnancy. If you're able to become pregnant, your provider will ask you to take a pregnancy test before starting treatment. In addition, you should use birth control during and for at least 2 months after the last dose of Opfolda and Pombiliti. Talk to your provider about your options if you're thinking about getting pregnant or are pregnant.
Headache and diarrhea were the most common side effects reported in people taking Pombiliti (cipaglucosidase alfa) with Opfolda (miglustat). And Pombiliti (cipaglucosidase alfa) infusions can also cause infusion-related side effects, such as muscle pain or spasms, tiredness, dizziness, fever, and chills. Some of the more serious side effect of this medication include heart or breathing problems. Talk with your provider about any concerns you have about Pombiliti (cipaglucosidase alfa) side effects.
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Pombiliti (cipaglucosidase alfa) dosage
Typical dosage for Pombiliti (cipaglucosidase alfa)
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