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).

Monthly appointment calendar
GIVLAARI is given once a month as a subcutaneous injection (under the skin) by a healthcare professional.

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.

Reduction of ALAS1 icon
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This leads to a reduction in levels of neurotoxins ALA and PBG.

Icon of neurotoxins ALA and PBG in a liver

ALA

PBG

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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

Down arrow representing 70% fewer porphyria attacks Down arrow representing 70% fewer porphyria attacks Patients on GIVLAARI had an average of 1.9 AHP attacks* compared to 6.5 in patients on 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

Down arrow representing 70% fewer days of hemin use Down arrow representing 70% fewer days of hemin use Patients on GIVLAARI had an average of 4.7 days of hemin use compared to 12.8 in patients on placebo
 

Important Safety Information

Do not use GIVLAARI if you have ever had a severe allergic reaction to GIVLAARI.

  • Tell your doctor or nurse right away if you experience any of the following signs or symptoms of a severe allergic reaction during treatment:

    • Swelling – mainly of the lips, tongue or throat which makes it difficult to swallow or breathe
    • Breathing problems or wheezing
    • Feeling dizzy or fainting
    • Rash or hives
    • Itching

    If you have a severe allergic reaction, your doctor or nurse will stop GIVLAARI treatment right away and you may need to take other medicines to control the symptoms.

  • Your doctor will check your liver function by doing blood tests:

    • Before you start using GIVLAARI
    • Once a month for the first 6 months of treatment
    • And when they think it is needed

    If these tests show abnormal results, your doctor or nurse will decide whether to temporarily interrupt or stop treatment with GIVLAARI.

  • Your doctor will check how your kidneys are working while you are using GIVLAARI.

  • GIVLAARI is given as an injection under the skin (called a "subcutaneous injection"). Reactions to this injection may happen during treatment with GIVLAARI.

    Tell your doctor or nurse right away if you experience any of the following symptoms of an injection site reaction during treatment: redness, pain, itchiness, rash, discoloration, or swelling around the injection site.

The most common side effects of GIVLAARI are nausea and injection site reactions. These are not all the possible side effects of GIVLAARI. Talk to your doctor about side effects that you experience. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-8 00-FDA -1088.


What is GIVLAARI?

GIVLAARI is a prescription medicine used to treat acute hepatic porphyria (AHP) in adults.


For additional information about GIVLAARI, please see full Prescribing Information.