Individual results may vary

"This was a body that I trusted and thought I knew well, but suddenly I felt betrayed and confused."
—Hannah, an Alnylam Patient Ambassador on GIVLAARI

Individual results may vary

"This was a body that I trusted and thought I knew well, but suddenly I felt betrayed and confused."
—Hannah, an Alnylam Patient Ambassador on GIVLAARI

What is acute hepatic porphyria (AHP)?

AHP is a family of rare, genetic diseases that can cause severe and potentially life-threatening attacks. Some people with AHP also have chronic, debilitating symptoms when they are not having an attack.

There are 4 types of AHP:

  1. 1.  Acute intermittent porphyria (AIP)
  2. 3.  Hereditary coproporphyria (HCP)
  3. 2.  Variegate porphyria (VP)
  4. 4.  ALAD-deficiency porphyria (ADP)
  1. 1.  Acute intermittent porphyria (AIP)
  2. 2.  Variegate porphyria (VP)
  3. 3.  Hereditary coproporphyria (HCP)
  4. 4.  ALAD-deficiency porphyria (ADP)

How AHP affects the body

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 aminolevulinic acid synthase 1 (ALAS1).

There are certain triggers that can activate ALAS1. Some known triggers include hormonal fluctuations, infection, stress, use of certain medications, alcohol consumption, and fasting/low-carbohydrate diets.

When ALAS1 is activated, the heme production process is unable to keep up. This causes toxic substances called aminolevulinic acid (ALA) and porphobilinogen (PBG) to build up. ALA and PBG are associated with attacks and other AHP symptoms. 

In AHP, when ALAS1 is activated in the liver the heme production process is unable to keep up.

Icon of toxins in a liver
Arrow

This causes toxins ALA and PBG to build up in the liver and be released throughout the body.

Icon of toxin buildup in a liver

ALA

PBG
Arrow

ALA and PBG are associated with attacks and other AHP symptoms.

GIVLAARI® (givosiran) reduces the amount of ALAS1 in the liver, which leads to a reduction in levels of the toxins ALA and PBG.
See how GIVLAARI® (givosiran) may help
See how GIVLAARI may help
 

IMPORTANT SAFETY INFORMATION 

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

GIVLAARI can cause:

  • Severe allergic reaction

    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.

  • Liver problems

    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.

  • Kidney problems

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

  • Injection site reactions

    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.

  • Increased blood homocysteine levels

    GIVLAARI may cause increased levels of homocysteine (a type of amino acid) in your blood. Your doctor will check your homocysteine levels before and during treatment by doing blood tests. If your levels are increased, your doctor may check your folate, vitamins B12 and B6, and tell you to take a vitamin B6 supplement.

  • Inflammation of the pancreas (pancreatitis)

    Cases of acute pancreatitis including some that were severe, have been reported in patients receiving GIVLAARI. If you have a severe case of acute pancreatitis your doctor or nurse will decide whether to temporarily interrupt or stop treatment with GIVLAARI.

What are the common side effects of GIVLAARI?

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® (givosiran)?

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


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