Individual results may vary
How
GIVLAARI® (givosiran)
may help
Individual results may vary
How
GIVLAARI® (givosiran)
may help
What is GIVLAARI?
GIVLAARI is a medicine used to treat acute hepatic porphyria (AHP) in adults. There are 4 types of AHP: acute intermittent porphyria (AIP), variegate porphyria (VP), hereditary coproporphyria (HCP), and ALA-dehydratase deficient porphyria (ADP).
How GIVLAARI works
In people with AHP, the heme production process in the liver does not work properly because of a genetic mutation. In the liver, this process is controlled by an enzyme called delta-aminolevulinate synthase 1 (ALAS1).
When ALAS1 is activated, the heme production process is unable to keep up. This causes neurotoxic substances called aminolevulinic acid (ALA) and porphobilinogen (PBG) to build up. ALA and PBG are associated with attacks and other AHP symptoms.
GIVLAARI reduces the amount of ALAS1 in the liver, which leads to a reduction in levels of the neurotoxins ALA and PBG.
In the liver, GIVLAARI reduces the level of ALAS1.


This leads to a reduction in levels of neurotoxins ALA and PBG.

ALA
PBG

ALA and PBG are associated with attacks and other AHP symptoms.
GIVLAARI results
In a 6-month study, GIVLAARI reduced AHP attacks* and days of hemin use compared to placebo
GIVLAARI was tested against a placebo in a clinical study of 94 adults with AHP who had 2 or more attacks* in the 6 months prior to the study. In the study, patients received either GIVLAARI or a placebo (an injection that did not contain any medication) once monthly over a 6-month period. During the study, patients taking GIVLAARI or placebo were able to use hemin if they experienced an attack.
Patients taking GIVLAARI experienced fewer AHP attacks* compared to those taking a placebo


*Attacks were defined as those that required hospitalization, urgent healthcare visit, or intravenous (IV) hemin at home.
Patients taking GIVLAARI needed less hemin to treat AHP attacks compared to those taking a placebo

