Loargys is a medicine used to treat people from 2 years of age with hyperargininaemia. Patients with hyperargininaemia are unable to break down the amino acid arginine because they lack the liver enzyme arginase 1. As a result, arginine accumulates in the body. This can cause problems with the nervous system, including seizures and stiffness in the legs. Hyperargininaemia is rare, and Loargys was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 14 July 2016. Further information on the orphan designation can be found on the EMA website: [ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1701](https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1701). Loargys contains the active substance pegzilarginase.
Therapeutic Indication
### Therapeutic indication Loargys is indicated for the treatment of arginase 1 deficiency (ARG1 D), also known as hyperargininemia, in adults, adolescents and children aged 2 years and older.
Therapeutic Area (MeSH)
ATC Code
A16AB24
ATC Item
pegzilarginase
Pharmacotherapeutic Group
Other alimentary tract and metabolism products
Active Substance (Summary)
INN / Common Names
| Substance | CAS | Monograph |
|---|---|---|
| Pegzilarginase | N/A | Pegzilarginase |
EMA Name
Loargys
Medicine Name
Loargys
Aliases
N/A