Givlaari (givosiran) is a prescription drug approved to treat acute hepatic porphyria (AHP). Givlaari comes as an injection given under the skin by a healthcare provider.

Specifically, Givlaari is used in adults to treat acute hepatic porphyria (AHP). AHP is a group of rare genetic conditions that cause a buildup of toxic substances which can lead to severe and potentially life threatening symptoms.

To learn more about Givlaari’s uses, see the “Uses for Givlaari” section.

Givlaari basics

Givlaari contains the active ingredient givosiran. It belongs to a group of drugs called aminolevulinate synthase 1-directed small interfering RNAs (siRNAs).

Givlaari is a brand-name medication. A generic version of the drug isn’t available currently.

Givlaari is used to treat AHP in adults.

Porphyria refers to a group of inherited conditions that cause problems with making a blood substance called heme. Your body uses heme to help carry oxygen and deliver it to cells.

With porphyrias, the body lacks certain enzymes needed to produce heme. This causes the buildup of substances called porphyrins, which are normally used to make heme. Buildup of these substances can lead to AHP symptoms, as well as porphyria “attacks,” or periods where symptoms worsen suddenly.

Porphyrin buildup leads to symptoms which vary depending on the specific porphyria. With AHP, symptoms can include:

How a drug works to treat a condition is also referred to as its mechanism of action. Givlaari’s mechanism of action to treat AHP is to reduce the buildup of porphyrins and porphyrin precursors, which helps lower the risk of AHP attacks and manages AHP symptoms.

Like most drugs, Givlaari may cause mild to serious side effects. The following lists contain some of the more common side effects Givlaari may cause, but they don’t include all possible side effects.

Keep in mind that side effects of a drug can depend on:

  • your age
  • other health conditions you have
  • other medications you take

Your doctor or pharmacist can tell you more about the potential side effects of Givlaari. They can also suggest ways to help reduce side effects.

Note: After the Food and Drug Administration (FDA) approves a drug, it tracks and reviews side effects of the medication. Sharing your experience helps make medications safer for everyone by giving doctors and researchers more information about how the drug works in real life. If you develop a side effect while taking Givlaari and want to tell the FDA about it, visit MedWatch or call 800-FDA-1088.

Mild side effects

Here’s a list of some of the mild side effects that Givlaari can cause. To learn about other mild side effects, talk with your doctor or pharmacist, or read Givlaari’s prescribing information.

Mild side effects of Givlaari that have been reported include:

  • nausea
  • injection site reactions, including any of the following at the place where Givlaari is injected:
    • pain
    • itching
    • swelling
    • skin redness or discoloration
  • skin rash or hives
  • fatigue
  • increased level of blood creatinine on a blood test
  • increased level of liver enzymes on a blood test

Mild side effects of many drugs may go away within a few days to a couple of weeks. But if they become bothersome, talk with your doctor or pharmacist.

Serious side effects

Serious side effects from Givlaari can occur, but they aren’t common. If you have serious side effects from Givlaari, call your doctor right away. If you think you’re having a medical emergency, you should call 911 or your local emergency number.

Serious side effects of Givlaari that have been reported include:

Allergic reaction

For some people, Givlaari can cause an allergic reaction.

In general, symptoms of an allergic reaction can be mild or serious.

What might help

If you have mild symptoms of an allergic reaction, such as a mild rash, call your doctor right away. They may suggest a treatment to manage your symptoms and can determine whether you should continue Givlaari treatment.

If you have symptoms of a severe allergic reaction, such as swelling or difficulty breathing, call 911 or your local emergency number right away. These symptoms could be life threatening and require immediate medical care.

If your doctor confirms you’ve had a serious allergic reaction to Givlaari, they may have you switch to a different treatment.

The Givlaari dosage your doctor prescribes will depend on several factors. These include:

  • your body weight in kilograms (kg)
  • any side effects you experience during Givlaari treatment

The following information describes dosages that are commonly used or recommended.Your doctor will determine the best dosage to fit your needs.

Form and strength

Givlaari comes as a liquid solution that’s given as an injection under your skin by a healthcare provider. It’s available in one strength: 189 milligrams in 1 milliliters of solution (mg/mL).

Recommended dosage

Your doctor will prescribe the dosage of Givlaari that’s right for you. Keep reading to learn more.

Dosage for AHP

For treatment of AHP, your doctor will likely prescribe a Givlaari dosage of 2.5 milligrams per kilogram of body weight (mg/kg) injected once per month. For reference, one kg equals about 2.2 pounds (lb).

For example, 68 kg is about 150 lb. If their doctor prescribes 2.5 mg/kg, their total Givlaari dose would be 170 mg.

Your doctor may prescribe a different Givlaari dosage in certain situations, including if you experience liver-related side effects during treatment. Talk with your doctor if you have questions about the dosage that’s right for you.

How it’s given

Your doctor will explain how Givlaari will be given to you. They’ll also explain how much you’ll be given and how often.

Givlaari is available as a liquid solution that’s given as an injection under your skin. You’ll receive these injections at a clinic or your doctor’s office.

About using Givlaari

The following are some common questions about using Givlaari.

  • What if I miss a dose of Givlaari? If you miss an appointment to receive an injection of Givlaari, call your doctor’s office as soon as possible to reschedule.
  • Will I need to use Givlaari long term? Givlaari is typically used as a long-term treatment. If you and your doctor determine that it’s safe and effective for your condition, you’ll likely use it long term.
  • How long does Givlaari take to work? Givlaari begins to work after you take your first dose. But it may take several weeks before you notice an improvement in your symptoms or a reduction in AHP attacks. Your doctor can provide more information on how they’ll monitor Givlaari’s effectiveness at treating your AHP symptoms.

Givlaari can interact with several other medications. It can also interact with certain supplements as well as certain foods.

Before taking Givlaari, talk with your doctor and pharmacist. Tell them about all prescription, over-the-counter, and other drugs you take. Also tell them about any vitamins, herbs, or supplements you use. Sharing this information can help you avoid potential interactions.

Interactions with medications, foods, and supplements

The following table highlights medications, supplements, and foods that can interact with Givlaari. This list does not contain all drugs that may interact with Givlaari. If you have questions about interactions that may affect you, ask your doctor or pharmacist.

Medications that can interact with Givlaari• certain medications for sleep, such as ramelteon (Rozerem) or • tasimelteon (Hetlioz)
• tizanidine (Zanaflex)
• duloxetine (Drizalma Sprinkle)
• dextromethorphan
• desipramine (Norpramin)
Vitamins or supplements that can interact with Givlaari• melatonin
Foods that can interact with Givlaari• caffeine

Givlaari and alcohol

Alcohol is not known to interact with Givlaari.

But alcohol may make your acute hepatic porphyria symptoms worse, including raising your risk for acute porphyria attacks. Because of this, your doctor may recommend that you avoid alcohol or limit the amount of alcohol you drink.

Whether you have health insurance or not, cost may be a factor when you’re considering Givlaari. What you’ll pay for Givlaari may depend on several things, such as your treatment plan and any costs to receive Givlaari injections from your doctor.

Here are a few things to consider regarding cost:

  • Savings program: If you have questions about how to pay for your prescription, talk with your doctor or pharmacist. A program called Alnylam Assist may also be available.

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The following is important information to consider and discuss with your doctor or pharmacist before using Givlaari.

Pregnancy and Givlaari

It’s not known if using Givlaari during pregnancy is safe. But there are risks with having untreated AHP while pregnant. (Givlaari is used for treating AHP.)

If you’re planning a pregnancy or can become pregnant, talk with your doctor about potential treatments for AHP during pregnancy. Together, you can consider the risks and benefits of your treatment options and decide whether you should take Givlaari during pregnancy.

Birth control and Givlaari

It’s not known whether Givlaari is safe to take during pregnancy. Therefore, if you’re sexually active and you or your partner can become pregnant, talk with your doctor about your birth control options while you’re using Givlaari.

Givlaari and breastfeeding

It’s not known whether Givlaari should be used while breastfeeding. If you’re currently breastfeeding or planning to do so, talk with your doctor before using this drug.

This section describes the warnings and precautions for Givlaari.

Before taking Givlaari, discuss your health history with your doctor. Givlaari may not be right for you if you have certain medical conditions or other factors affecting your health. Be sure to talk with your doctor if any of the following apply to you:

Note: For more information about the potential negative effects of Givlaari, see the “Side effects of Givlaari” section.

Currently, there aren’t other drugs similar to Givlaari which are approved for treating AHP. But there are other medications used to treat AHP attacks, as well as help manage symptoms of AHP.

The following drugs may be used to treat AHP attacks or manage AHP symptoms:

  • hemin injection (Panhematin)
  • medications for managing blood pressure
  • medications for managing pain
  • medications for managing nausea

Disclaimer: Healthline has made every effort to make certain that all information is factually correct, comprehensive, and up to date. However, this article should not be used as a substitute for the knowledge and expertise of a licensed healthcare professional. You should always consult your doctor or another healthcare professional before taking any medication. The drug information contained herein is subject to change and is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects. The absence of warnings or other information for a given drug does not indicate that the drug or drug combination is safe, effective, or appropriate for all patients or all specific uses.