DISEASE INDICATIONS: Acute Hepatic Porphyria (AHP)
MANUFACTURER: Alnylam Pharmaceuticals
USAGE: Subcutaneous
MEDICINE APPROVED BY:
European Medical Agency (EMA)
Food and Drug Administration (FDA)
Pharmaceuticals and Medical Devices Agency (PMDA)
Givlaari (givosiran) is a synthetic small interfering RNA medication given as a monthly subcutaneous injection to prevent attacks of acute intermittent porphyria by reducing the production of the liver enzyme ALAS1, which leads to toxic heme precursors accumulation.