Givlaari is a medicine for treating acute hepatic porphyria in patients aged 12 years or over. Acute hepatic porphyria is a rare genetic condition in which the liver cannot properly produce a substance called haem. As a result, substances used to make haem build up in the body, causing attacks of severe abdominal pain, vomiting and nervous system disorders. Acute hepatic porphyria is rare, and Givlaari was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 29 August 2016. Givlaari contains the active substance givosiran.
Therapeutic Indication
### Therapeutic indication Treatment of acute hepatic porphyria (AHP) in adults and adolescents aged 12 years and older.
Therapeutic Area (MeSH)
ATC Code
A16AX16
ATC Item
givosiran
Pharmacotherapeutic Group
Various alimentary tract and metabolism products
Active Substance (Summary)
INN / Common Names
| Substance | CAS | Monograph |
|---|---|---|
| Givosiran | N/A | 吉伏司瑞 |
EMA Name
Givlaari
Medicine Name
Givlaari
Aliases
N/A