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 Brands | Opfolda |
|---|---|
| Drug Class | Enzyme stabilizer |
| Controlled Substance Classification | Not a controlled medication |
| Generic Status | No lower-cost generic available |
| Availability | Prescription only |
Opfolda (miglustat) dosage
Typical dosage for Opfolda (miglustat)
What are alternatives to Opfolda (miglustat)?
Opfolda (miglustat) images
