Givlaari (givosiran) is a medication that treats acute hepatic porphyria (AHP), a rare, genetic disease that causes painful attacks on your body. It's a once-monthly injection that your provider gives you.
Adults with acute hepatic porphyria (AHP)
Givlaari (givosiran) is a small interfering ribonucleic acid (siRNA). This molecule targets a protein in your liver called aminolevulinate synthase (ALAS1). In AHP, ALAS1 becomes too active in the liver, which causes toxic substances to build up in the body. Givlaari (givosiran) works by lowering the amount of ALAS1 in your liver. This causes the levels of toxic substances to drop, which helps to treat symptoms of AHP.
Source: DailyMed
Side effects that you should report to your care team as soon as possible:
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
Hasn't been well studied in children under 18 years old or adults 65 years and older
Needs to be given by a healthcare provider
Brand name only, which can be expensive
Make sure to mark your calendar with your appointments to see your provider and receive your dose of Givlaari (givosiran) so you don't miss any doses. Also mark when you're supposed to get lab tests done so you can be properly monitored.
Keep track of how you feel in between Givlaari (givosiran) injections to see if there's improvement in your symptoms over time. This way, you can compare your progress from month to month, and discuss with your provider if you think this medication isn't working well for you.
Givlaari (givosiran) can interact with certain medications. Make sure to tell your provider about all the medications you're taking before starting Givlaari (givosiran). Don't start any new medications while taking Givlaari (givosiran) without talking to your provider or pharmacist first.
Givlaari (givosiran) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Although rare, severe allergic reactions to Givlaari (givosiran) are possible, including life-threatening reactions like facial swelling, shock, and anaphylaxis (closing of the throat). If you notice dizziness, a choking sensation, difficulty breathing, wheezing, swelling of the lips or tongue, skin flushing, or rash after receiving Givlaari (givosiran), call 911 right away.
Givlaari (givosiran) might cause liver and kidney problems. You'll need to get regular blood tests done to make sure your liver and kidney are working properly. If your lab results show signs of liver or kidney problems, your provider might temporarily stop your Givlaari (givosiran) treatment so they can figure out whether the medication is causing these problems. Get medical attention right away if you notice signs of liver problems (e.g., yellowing of your skin or the whites of your eyes, fatigue, low appetite, nausea, stomach discomfort, confusion) or kidney problems (e.g., changes in how much you urinate, muscle cramps, weakness, swelling, dry skin).
Givlaari (givosiran) can raise your blood homocysteine levels. Homocysteine is an amino acid your body makes. It's not clear how high homocysteine levels from the medication affects your health, but we know that too much homocysteine in your blood can generally damage your blood vessels. Your provider will measure your homocysteine levels before and during treatment with Givlaari (givosiran). If your homocysteine levels are high, your provider might recommend you start taking vitamin supplements, such as vitamin B6 or a multivitamin.
Givlaari (givosiran) is only available as an injection that's administered by a healthcare provider.
The dose is based on your body weight. In general, the typical dose is 2.5 mg/kg of body weight injected under the skin once per month. Your dose might differ if you're having side effects affecting your liver.
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