Elfabrio is a medicine used in adults to treat Fabry disease, a rare inherited disorder. Patients with Fabry disease do not have enough of an enzyme, called alpha-galactosidase A, that normally breaks down a fatty substance called globotriaosylceramide (Gb3). If the enzyme is not present, Gb3 cannot be broken down and it builds up in the organs, such as the kidney and heart, causing kidney failure and heart problems. Elfabrio contains the active substance pegunigalsidase alfa.
Therapeutic Indication
### Therapeutic indication Elfabrio is indicated for long-term enzyme replacement therapy in adult patients with a confirmed diagnosis of Fabry disease (deficiency of alpha-galactosidase).
Therapeutic Area (MeSH)
ATC Code
A16AB20
ATC Item
pegunigalsidase alfa
Pharmacotherapeutic Group
Other alimentary tract and metabolism products
Active Substance (Summary)
INN / Common Names
| Substance | CAS | Monograph |
|---|---|---|
| pegunigalsidase alfa | N/A | Pegunigalsidase alfa |
| Pegunigalsidase alfa | N/A | Pegunigalsidase alfa |
EMA Name
Elfabrio
Medicine Name
Elfabrio
Aliases
N/A
