Opfolda is a medicine used in the treatment of adults with late-onset Pompe disease (acid alpha-glucosidase \[GAA\] deficiency), an inherited disorder in which patients have breathing difficulties and muscle weakness. It is used in combination with another medicine, cipaglucosidase alfa. Opfolda is a ‘hybrid medicine’. This means that it is similar to a ‘reference medicine’ containing the same active substance, but it is available as capsules of a different strength.The reference medicine for Opfolda is Zavesca. Opfolda contains the active substance miglustat.
Therapeutic Indication
### Therapeutic indication Opfolda (miglustat) is an enzyme stabiliser of cipaglucosidase alfa long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid ?- glucosidase \[GAA\] deficiency).
Therapeutic Area (MeSH)
ATC Code
A16AX06
ATC Item
miglustat
Pharmacotherapeutic Group
Other alimentary tract and metabolism products
Active Substance (Summary)
INN / Common Names
| Substance | CAS | Monograph |
|---|---|---|
| miglustat | N/A | 麦格司他 |
EMA Name
Opfolda
Medicine Name
Opfolda
Aliases
N/ANo risk management plan link.
