Opfolda
Opfolda (miglustat) is an enzyme stabilizer. It's used along with another medication called Pombiliti (cipaglucosidase alfa-atga) to treat Pompe disease in adults. Opfolda (miglustat) protects Pombiliti (cipaglucosidase alfa-atga) from being broken down in the body and is taken by mouth an hour before a Pombiliti (cipaglucosidase alfa-atga) infusion. You'll typically receive this treatment every 2 weeks, as directed by your provider. The most common side effects of this combination are headache and diarrhea.
What is Opfolda (miglustat)?
What is Opfolda (miglustat) used for?
- Late-onset Pompe disease in adults weighing at least 88 lbs (40 kg) - in combination with Pombiliti (cipaglucosidase alfa-atga)
How Opfolda (miglustat) 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. Opfolda (miglustat) is an enzyme stabilizer. It attaches to Pombiliti to prevent it from breaking down in the body. This allows Pombiliti enough time to get absorbed into the cells, where it can do its job of breaking down glycogen.
Drug facts
Common questions about Opfolda (miglustat)
Pompe disease is a genetic condition. People with this condition don't have enough of an enzyme (protein) called acid alpha-glucosidase (GAA). Normally, this enzyme provides energy to your cells by breaking down the stored form of sugar called glycogen. Without GAA, the glycogen can't be broken down, which can cause muscle weakness. While Pompe disease can affect your whole body, your heart and skeletal muscles are the most affected, which can be life-threatening.
Opfolda (miglustat) is available as capsules. Your provider will determine your dose based on body weight and if you have kidney problems. Opfolda (miglustat) is taken by mouth once every 2 weeks on an empty stomach, an hour before the Pombiliti (cipaglucosidase alfa-atga) infusion. You won't be able to eat or drink anything 2 hours before and 2 hours after taking this medication]. But you can take Opfolda (miglustat) with an unsweetened drink, like water or tea or coffee with no cream, sugar, or other sweeteners.
No, Opfolda (miglustat) and Pombiliti (cipaglucosidase alfa-atga) 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.
If you missed your Opfolda (miglustat) dose, your Pombiliti (cipaglucosidase alfa-atga) infusion will need to be rescheduled. It's very important to not miss any doses because doing so can cause your Pompe disease symptoms to worsen or come back. Mark the days and times of when you need to see your provider and receive Pombiliti (cipaglucosidase alfa-atga) so you know when to stop eating and drinking and take Opfolda (miglustat). If you miss a Opfolda (miglustat) dose, call your provider right away to reschedule your treatment.
In clinical studies, people taking Opfolda (miglustat) with Pombiliti (cipaglucosidase alfa-atga) most commonly reported headache and diarrhea. Other less common side effects include dizziness, shortness of breath, bloating, fever, and rash.
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Opfolda (miglustat) dosage
Typical dosage for Opfolda (miglustat)
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