Individual results may vary

Lina, patient with AIP

Individual results may vary

Lina, patient with AIP

GIVLAARI® (givosiran) study results

In a 6-month study, GIVLAARI reduced AHP attacks and days of hemin use compared with placebo

In the study, 48 patients received GIVLAARI and 46 patients received placebo (an injection that did not contain any medicine) once monthly over a 6-month period. During the study, patients were able to use hemin if they experienced an attack. Using hemin to prevent an attack was not allowed during the study.

Patients taking GIVLAARI experienced fewer AHP attacks on average compared with 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 with 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 on average to treat AHP attacks compared with 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 with 12.8 in patients on placebo

Fewer patients experienced AHP attacks over 36 months

After the 6-month study, all 93 eligible patients who remained in the study received GIVLAARI once a month. The graph below shows the 48 patients who were treated with GIVLAARI in the 6-month study and continued on in the study. Over time, fewer patients had attacks.

Figure showing percentage of study patients who experienced AHP attacks at baseline and up to 36 months

Attacks measured in the study were defined as those that required hospitalization, urgent healthcare visits, or IV hemin at home.

These results were observed in the clinical trial. Keep in mind that everyone responds to GIVLAARI differently.

GIVLAARI safety profile

Safety during the first 6 months of the study

  • In the first 6 months of the study, 1 patient taking GIVLAARI stopped treatment due to changes in liver function. No patients taking placebo stopped treatment

The most common side effects in patients treated with GIVLAARI compared with those taking placebo in the first 6 months of the study were:

  GIVLAARI
(48 patients)		 Placebo
(46 patients)
Nausea 27% 11%
Injection site reactions 25% 0%
Rash 17% 4%
Changes in kidney function 15% 4%
Changes in liver function 13% 2%
Fatigue 10% 4%

Safety after the first 6 months

  • The most common treatment-related side effects (in ≥10% of patients) were injection site reactions (32% [30/94] patients), nausea (21% [20/94]), and fatigue (14% [13/94])
  • Serious side effects were reported in 7 patients (7% of patients). Serious side effects that were reported in 2 or more patients were increased blood homocysteine (2 patients) and serious side effects related to elevated liver function tests (increased liver enzymes and an abnormal liver function test in 1 patient each)
  • During this period, there was 1 death from an aortic dissection unrelated to GIVLAARI treatment
  • After the initial 6-month study, 3 patients stopped treatment due to side effects. One stopped because of a severe allergic reaction, and the other 2 stopped due to increased blood homocysteine
  • During this period, increased blood homocysteine levels were found in 15 of 93 patients (16%)

     

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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.

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.