Givlaari Givlaari

Generic name:

GIVOSIRAN SODIUM

Company:

ALNYLAM PHARMS INC

FDA approval date:

11/20/2019

Specific treatment:

GIVLAARI is an aminolevulinate synthase 1-directed small interfering
RNA indicated for the treatment of adults with acute hepatic porphyria
(AHP).

Mechanism of Action:

Givosiran is a double-stranded small interfering RNA that causes degradation of aminolevulinate
synthase 1 (ALAS1) mRNA in hepatocytes through RNA interference, reducing the elevated levels of liver
ALAS1 mRNA. This leads to reduced circulating levels of neurotoxic intermediates aminolevulinic acid
(ALA) and porphobilinogen (PBG), factors associated with attacks and other disease manifestations of
AHP.

Dosage Form/Route:

The recommended dose of GIVLAARI is 2.5 mg/kg once monthly by
subcutaneous injection.

Side Effects:

The most common adverse reactions (≥20% of patients) included
nausea and injection site reactions.