Pombiliti is a medicine used to treat adults with late-onset Pompe disease (acid alpha-glucosidase \[GAA\] deficiency), an inherited disorder in which patients have breathing difficulties and muscle weakness. Pombiliti is used in combination with another medicine, miglustat. Pombiliti contains the active substance cipaglucosidase alfa.
Therapeutic Indication
### Therapeutic indication Pombiliti (cipaglucosidase alfa) is a long-term enzyme replacement therapy used in combination with the enzyme stabiliser miglustat for the treatment of adults with late-onset Pompe disease (acid ?-glucosidase \[GAA\] deficiency).
Therapeutic Area (MeSH)
ATC Code
A16AB
ATC Item
酶类
Pharmacotherapeutic Group
Other alimentary tract and metabolism products
Active Substance (Summary)
INN / Common Names
| Substance | CAS | Monograph |
|---|---|---|
| Cipaglucosidase alfa | N/A | Cipaglucosidase alfa |
EMA Name
Pombiliti
Medicine Name
Pombiliti
Aliases
N/A