What is Givlaari?
Givlaari is a treatment that reduces the number of acute attacks in acute hepatic porphyria and helps to use intravenous heme. Acute hepatic porphyria (AHP) is a rare genetic disease that can cause severe attacks of the nervous system. Symptoms include stomach pain, nausea and vomiting, numbness, or mental status changes.A genetic mutation causes acute hepatic porphyria, which is a disorder that disrupts normal heme production. Heme is a component of hemoglobin. This disruption causes the liver to accumulate toxic compounds called porphyrins, which then enter the bloodstream, causing symptoms of AHP. The production of heme in the liver is controlled by aminolevulinic synthase 1, or ALAS1.Givlaari, an aminolevulinate synase-1-directed small interfering (siRNA) enzyme, works by reducing the amount of ALAS1 within the liver. This leads to a decrease in toxic compounds in the body and helps improve AHP symptoms.Givlaari, a subcutaneous injectable, is typically given once a month.The disorder is more prevalent in women who are pregnant.
Warnings
Your next dose could be delayed, depending on the results of your blood tests.
Before you take this drug
Givlaari should not be used if you have an allergy to any of its inactive ingredients or the product itself.
Pregnancy
Notify your doctor immediately if you plan on becoming pregnant. Givlaari is not known to harm a baby in utero. Acute hepatic porphyria can cause serious health problems for both mother and child. This disorder can be treated without causing any harm to the child.
Breastfeeding
Breastfeeding may not be recommended while taking Givlaari. You should ask your doctor how to best feed your baby while taking Givlaari
How to take Givlaari?
Please read the instructions and all the guides that come with your medication. Sometimes your doctor will change the dose.Givlaari injections are usually given once a month, under the skin. This injection will be given by a healthcare provider.The Givlaari dosage is based on your weight. Your dosage requirements could change if you gain or lose weight.Before and during the treatment with Givlaari, you will be required to undergo frequent blood tests in order to monitor your liver function. The results may delay your next dose.You may also be required to have your kidney function checked.
Details on dosage
Givlaari should be administered at a dose of 2.5 mg/kg via subcutaneous injection once a month. Givlaari dosage is determined by actual body weight.Givlaari is injected into the abdomen, upper arms (back or side), or thighs. Rotate injection sites.Injections should not be administered to areas with reddened or inflamed tissue or that are swollen or inflamed.Avoid injecting in a circle of 5 cm around the navel if you are injecting into your abdomen.If you need to inject more than once for one dose of Givlaari, then the sites must be separated by at least 2cm from each other.
What happens if I miss the dose?
Do not use two doses at once. Never use two doses in one go.
What happens if you overdose?
Givlaari will not cause an overdose if it is administered by a health professional in a medical environment.
Side effects of Givlaari
If you experience any of the following symptoms of an allergic reaction, seek immediate medical attention: hives, difficulty breathing, swelling of your lips, face, tongue, or throat.If you experience certain side effects, your injections could be delayed or discontinued permanently.Consult your doctor immediately if you experience pain or tenderness in the upper abdomen, pale stools or dark urine, nausea or vomiting, or yellow skin or eyes. These symptoms could indicate a serious liver condition.Consult your doctor immediately if you experience bloody urine or a change in the amount or frequency of urine. You may also notice an increase in blood pressure, an increase in thirst, decreased appetite, pain in the lower back or sides, nausea, swelling in the fingers or legs, difficulty breathing, unusual fatigue or weakness, weight gain, or even nausea. These symptoms could indicate a serious kidney issue.If you experience redness, pain, swelling, or burning at the injection site, call your doctor immediately.You may need to take vitamin supplements (e.g., vitamin B6) if you have a blood problem. Vitamin supplements (e.g., vitamin B6 supplements) may be prescribed to treat this problem.
Some of the common side effects that Givlaari can cause include:
- Nausea;
- Pain, itching, or swelling at the injection site.
There may be other side effects. For medical advice on side effects, call your doctor. The FDA can be contacted at 1-800-FDA-1088 to report side effects.
Interaction with other drug
It is sometimes not safe to take certain medications together. Some drugs may affect the blood levels of drugs that you are taking, which can increase side effects and make them less effective.Givlaari is not usually recommended with the following medications, but it may be necessary in certain cases. Your doctor may alter the dosage or frequency of use of one or both medications if both are prescribed.
