What Is Givlaari?
Givlaari (givosiran) is an injectable prescription medication used to treat adults with acute hepatic porphyria (AHP). AHP is a group of rare genetic conditions in which there’s not enough of a special liver enzyme that converts porphyrin to heme in the body. This creates a buildup of toxins in the body, which can lead to painful brain and nerve attacks and sometimes skin conditions like a rash or excessive hair growth.
Givlaari belongs to a group of drugs called aminolevulinate synthase 1-directed small interfering RNA. Givlaari works by preventing the buildup of toxic substances in the liver that can cause AHP attacks.
Your healthcare provider will inject Givlaari under your skin (subcutaneously) once per month.
Generic Name: Givosiran
Brand Name: Givlaari
Drug Availability: Prescription
Therapeutic Classification: Aminolevulinate synthase 1-directed small interfering RNA
Available Generically: No
Controlled Substance: N/A
Administration Route: Subcutaneous
Active Ingredient: Givosiran
Dosage Form: Injectable solution
What Is Givlaari Used For?
The Food and Drug Administration (FDA) has approved Givlaari (givosiran) to treat adults with acute hepatic porphyria (AHP). AHP is a group of rare genetic disorders that affect the production of heme in your liver. Heme is an essential compound that your body uses for many important functions. In people with AHP, heme production becomes altered, which can cause a buildup of toxic substances. These toxins can lead to an AHP attack.
Symptoms of an AHP attack include:
- Abdominal pain
- Fast heartbeat
- High blood pressure
- Depression or anxiety
- Tingling or numbness
- Sun sensitivity
Givlaari can help reduce the number of AHP attacks you experience. Givlaari can also decrease how often you need to use Panhematin (hemin)—an intravenous (IV) medicine used to treat AHP attacks.
How to Take Givlaari
Your healthcare provider will inject Givlaari under your skin (subcutaneously) once a month. Givlaari can be injected into your stomach, upper arm or thigh. Be sure to choose a location without any scarring, redness, swelling or irritation.
Your healthcare provider will store Givlaari at their office.
How Long Does Givlaari Take to Work?
Givlaari takes time to work. In clinical studies, participants who received Givlaari experienced significantly fewer AHP attacks than those who received a placebo (an injection without any medication) during six months of treatment. It’s important to keep a daily journal of how you are feeling and any symptoms you notice. This will help track your progress over time. Be sure to share these findings with your healthcare provider at each appointment.
What Are the Side Effects of Givlaari?
This is not a complete list of side effects and others may occur. A medical professional can advise you on side effects. If you experience other effects, contact your pharmacist or a medical professional. You may report side effects to the FDA at www.fda.gov/medwatch or 800-FDA-1088.
Common Side Effects
You may experience side effects from Givlaari. Be sure to let your healthcare provider know if you develop any reactions. Common side effects include:
- Injection site reactions (e.g., redness, pain, itching, rash, changes in skin color or swelling)
Some people may also develop high homocysteine blood levels. Homocysteine is a naturally occurring substance in your body, but health problems can occur if levels become too high. Your healthcare provider will monitor your homocysteine levels with a blood test before you start Givlaari and during treatment.
Severe Side Effects
Rarely, Givlaari may cause severe side effects. Call your healthcare provider right away if you develop any serious reaction. Call 911 if your symptoms feel life-threatening or if you think you’re having a medical emergency. Serious side effects and their symptoms include:
- Allergic reactions, including a severe form called anaphylaxis: Seek medical care if you develop a rash, wheezing, trouble breathing, or swelling of your face, mouth, tongue, or throat.
- Liver problems: Your healthcare provider will monitor your liver function with a blood test before you start Givlaari and during treatment. Signs of liver problems include dark-colored urine, tiredness, loss of appetite, nausea or vomiting, stomach pain, light-colored stools and yellowing of your skin or the white parts of your eyes.
- Kidney problems: Let your healthcare provider know if you are unable to urinate or notice a decrease in the amount that you urinate, blood in your urine, or a big weight gain.
Report Side Effects
Givlaari may cause other side effects. Call your healthcare provider if you have any unusual problems while taking this medication.
If you experience a serious side effect, you or your provider may send a report to the FDA’s MedWatch Adverse Event Reporting Program or by phone (800-332-1088).
Dosage: How Much Givlaari Should I Take?
Your healthcare provider will choose and administer your dosage of Givlaari.
- Pregnancy: There is no information on the safety of using Givlaari during pregnancy in humans. Pregnant animals that were given high doses of Givlaari developed complications. You and your healthcare must discuss the risks and benefits of using Givlaari during pregnancy.
- Breastfeeding: Information about Givlaari use during breastfeeding is lacking. Talk with your healthcare provider about the best way to feed your baby.
- Children: Givlaari has not been studied in children with AHP. The FDA has only approved Givlaari to treat adults with AHP.
- Adults over the age of 65 years: There is not enough information available to know if people over the age of 65 will respond differently to Givlaari compared with younger adults.
Givlaari is administered (given) once a month at your healthcare provider’s office. It’s important to schedule your appointments ahead of time to ensure that you stay on track. If you miss a dose, call your healthcare provider to schedule another appointment as soon as possible. Schedule your next dose one month after you receive your missed dose. Some people find it helpful to schedule reminders in their calendars or phones.
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Your doctor will check your progress closely while you are receiving this medicine. This will allow your doctor to see if the medicine is working properly and to decide if you should continue to receive it. Blood tests may be needed to check for unwanted effects.
This medicine may cause serious allergic reactions, including anaphylaxis, which can be life-threatening and require immediate medical attention. Tell your doctor right away if you have a rash, itching, trouble breathing, trouble swallowing, or any swelling of your hands, face, or mouth while you are using this medicine.
Check with your doctor right away if you have pain or tenderness in the upper stomach, pale stools, dark urine, loss of appetite, nausea, vomiting, or yellow eyes or skin. These could be symptoms of a serious liver problem.
Check with your doctor right away if you have bloody urine, a decrease in frequency or amount of urine, an increase in blood pressure, increased thirst, loss of appetite, lower back or side pain, nausea, swelling of the face, fingers, or lower legs, trouble breathing, unusual tiredness or weakness, vomiting, or weight gain. These could be symptoms of a serious kidney problem.
Call your doctor right away if you have redness, burning, swelling, or pain at the injection site.
This medicine may increase homocysteine (an amino acid) levels in your blood. You may receive vitamin supplements (eg, vitamin B6 supplement) to treat this blood problem.
Do not take other medicines unless they have been discussed with your doctor. This includes prescription or nonprescription (over-the-counter [OTC]) medicines and herbal or vitamin supplements.
What Are Reasons I Shouldn’t Take Givlaari?
Do not take Givlaari if you are allergic to givosiran (the active ingredient in Givlaari). Let your healthcare provider know about all your medical conditions and allergies so they can decide if Givlaari is the best treatment for you.
What Other Medications Interact With Givlaari?
Many medications may interact with Givlaari. Be sure to let your healthcare provider know about all the medicines that you take, including prescription and over-the-counter (nonprescription) products. This includes supplements and products containing herbs or plants.
Givlaari can raise the levels of certain medications and increase your chance of developing side effects. Let your healthcare provider know if you take:
- Azilect (rasagiline)
- Cerdelga (eliglustat)
- DayVigo (lemborexant)
- Esbriet (pirfenidone)
- Juxtapid (lomitapide)
- Lotronex (alosetron)
- Mellaril (thioridazine)
- Orap (pimozide)
- Rapamune (sirolimus)
- Soltamox (tamoxifen)
- Theo-24 (theophylline)
- Ubrelvy (ubrogepant)
- Zanaflex (tizanidine)
Many other medications may interact with Givlaari. Always keep an up-to-date list of all the medicines you take, and let your healthcare provider and pharmacist know any time there are changes.
What Medications Are Similar?
Givlaari is unique in the way it works. It is the only medication in its class (aminolevulinate synthase 1-directed small interfering RNA) used to prevent AHP attacks.
Frequently Asked Questions
What is Givlaari used for?
Givlaari is used to treat adults with acute hepatic porphyria (AHP). Givlaari helps prevent AHP attacks.
How does Givlaari work?
People with AHP produce abnormally high amounts of certain liver toxins. Givlaari helps prevent AHP attacks by decreasing the production of these toxic substances.
What are the side effects of Givlaari?
The most common side effects of Givlaari are nausea and injection site reactions (e.g., redness, pain, itching, swelling, rash).
How is Givlaari administered?
Givlaari is an injectable medication that your healthcare provider injects under your skin (subcutaneously). Givlaari is administered once a month.
How Can I Stay Healthy While Taking Givlaari?
The uncertainty of when an AHP attack might strike can leave you feeling stressed and apprehensive about making plans. Fortunately, Givlaari can help reduce attacks and let you focus on the things you enjoy.
Identifying triggers that can lead to an attack is another important part of managing your condition. Certain medications, alcohol, tobacco use, fasting or extreme dieting, and stress can all cause an AHP attack. Stopping use of alcohol and/or quitting tobacco can be helpful.
A registered dietitian nutritionist (RD or RDN) can discuss with you foods that will support your condition. Talk with your healthcare provider about your risks and discuss any changes in your diet and lifestyle that you can make to help avoid AHP triggers.
Verywell Health’s drug information is meant for educational purposes only and is not intended as a replacement for medical advice, diagnosis, or treatment from a healthcare professional. Consult your healthcare provider before taking any new medication(s). IBM Watson Micromedex provides some of the drug content, as indicated on the page.