Pronunciation: ul-toe-meer-is
Generic name: ravulizumab
Dosage form: injection for intravenous or subcutaneous use
Drug class: Selective immunosuppressants

Medically reviewed by Carmen Pope, BPharm. Last updated on Apr 2, 2024.

What is Ultomiris?

Ultomiris (ravulizumab-cwvz) is an injectable complement inhibitor that may be used to:

• treat paroxysmal nocturnal hemoglobinuria (PNH) in adults and children aged one month and older
• inhibit complement-mediated thrombotic microangiopathy (TMA) in adults and children aged one month and older with atypical hemolytic uremic syndrome (aHUS) (does not include Shiga toxin E. coli-related hemolytic uremic syndrome [STEC-HUS])
• treat generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) antibody-positive
• treat neuromyelitis optica spectrum disorder (NMOSD) in adults who are anti-aquaporin-4 (AQP4) antibody positive.

Ultomiris works by inhibiting the activity of the complement system, which is part of the immune system. It specifically binds to complement protein C5 with high affinity, inhibiting its cleavage to C5a (a proinflammatory toxin) and C5b (which initiates the membrane attack complex (MAC or C5b-9) which leads to osmolysis and the rupture of the cell wall.  Ultomiris prevents complement-mediated red blood cell rupture in patients with PNH and complement-mediated thrombotic microangiopathy (TMA) in patients with aHUS. The exact way Ultomiris works for gMG or NMOSD is unknown but is thought to involve decreased deposition of C5b-9.

Ultomiris is a monoclonal antibody. Monoclonal antibodies are man-made antibodies that fight antigens (harmful substances) in the body. It affects your immune system and may lower the ability of your body to fight infections.

Ultomiris is available only under a special program called the Ultomiris and Soliris REMS. Before you can receive it, your healthcare provider must:

• enroll in the REMS program
• counsel you about the risk of serious meningococcal infections
• give you information about the signs and symptoms of serious meningococcal infection
• make sure that you are vaccinated against serious infections caused by meningococcal bacteria and that you receive antibiotics if you need to start Ultomiris right away and you are not up to date on your vaccines
• give you a Patient Safety Card about your risk of meningococcal infection.

Ultomiris was FDA-approved on December 21, 2018.

Related/similar drugs

Rystiggo, Vyvgart, Zilbrysq, pyridostigmine, Mestinon, neostigmine, Soliris

Warnings

Ultomiris affects your immune system. You may get infections more easily, even serious or fatal infections, such as meningitis. Call your doctor if you have a fever, flu-like symptoms, muscle pain, headache, confusion, neck or back stiffness, vomiting, rash, or if your eyes are more sensitive to light.

You will need to receive certain vaccinations, including vaccinations to protect against meningococcal disease, at least 2 weeks before you start using Ultomiris, unless the risks of delaying Ultomiris outweigh the risks of a serious infection.

Ultomiris comes with a Patient Safety Card listing symptoms of meningococcal infection. Keep this card with you at all times while using Ultomiris and for at least 8 months after your last dose. Your infection risk could last for several months after you stop using this medicine.

Some people may have an increased risk of gonorrhea (a sexually transmitted disease) while using this medicine. Talk with your doctor about safe ways to keep from getting an infection during sex.

Before taking

Before you receive Ultomiris, tell your healthcare provider about all of your medical conditions, including if you:

• are allergic to ravulizumab or Ultomiris
• have a meningococcal infection (such as meningitis or sepsis), or if you are not currently vaccinated against meningitis (unless the risks of delaying treatment outweigh the risks of developing meningitis). You will need to receive a vaccine to protect against meningococcal infections at least 2 weeks before you start using Ultomiris. If your child is treated with this medicine, make sure he or she is vaccinated against pneumonia and influenza type B (Hib). If you need to start receiving this medicine before you are vaccinated, you may be given antibiotic medicine to take during the first 2 weeks of Ultomiris treatment.
• have recently had any symptoms of an infection (fever, chills, or flu-like symptoms) or recently have had meningitis
• are pregnant or intend to become pregnant
• are breastfeeding or intend to breastfeed.

Ultomiris is not approved for use in PNH by anyone younger than 1 month old. For other indications (such as gMG and NMOSD), it is not approved for use in children. Subcutaneous administration of Ultomiris has not been evaluated and is not approved for use in children.

Pregnancy

It is not known if Ultomiris will harm your unborn baby. Tell your doctor if you are pregnant. It is not known whether ravulizumab will harm an unborn baby. However, having PNH during pregnancy may cause complications in the baby or the mother, including blood clots, infections, bleeding, miscarriage, premature delivery, or death. The benefit of treating PNH may outweigh any risks to the baby or the mother.

Breastfeeding

It is not known if Ultomiris passes into your breast milk. You should not breastfeed during treatment and for 8 months after your final dose.

How is Ultomiris administered?

Ultomiris is administered either as an intravenous (into a vein) infusion by a healthcare provider or self-administered by subcutaneous (under the skin) injection through an on-body injector.

Intravenous administration

Ultomiris is given by infusion. The time it takes to administer the infusion ranges from a minimum of 0.4 hours to 1.4 hours and depends on body weight and dosage. Your dose may change if you gain or lose weight. See the product information for minimum infusion times.

• After each infusion, you will be watched closely for at least 1 hour to make sure you do not have an allergic reaction.
• You will usually receive a starting dose of Ultomiris, followed 2 weeks later by another infusion.
• The infusion is then usually repeated every 8 weeks in adults.

In children aged 1 month and older with PNH or aHUS, the infusion is usually given every 4 weeks or every 8 weeks, depending on their weight, starting 2 weeks after the starting dose.

Subcutaneous administration

For adults with PNH or aHUS, Ultomiris may be administered subcutaneously (SC) through an on-body injector. Usually, the very first dose is given as an intravenous infusion by your healthcare provider.

• Do not attempt to self-administer Ultomiris until you have received training from your healthcare provider about the correct way to prepare and give your SC injection. Use it exactly as your healthcare provider tells you to.
• You will need 2 on-body delivery systems (each containing 1 on-body injector and 1 prefilled cartridge) for a full SC dose, and each injection will take about 10 minutes.
• You or your caregiver can administer the injections at the same time or one after the other into your stomach (abdomen), thigh, or upper arm.

Subcutaneous injections of Ultomiris are usually given weekly.

• After each administration, you should monitor yourself for allergic and infusion-related reactions for at least 1 hour.
• If you are changing between IV and SC Ultomiris, then you should receive your first dose of SC Ultomiris 8 weeks after your last IV dose.
• If you've been using another drug called eculizumab (Soliris), you will need to wait 2 weeks after your last dose of eculizumab before starting treatment with Ultomiris.

Stopping treatment

Your healthcare provider will tell you how long you should stay on Ultomiris.

If you have PNH and you stop receiving Ultomiris, your healthcare provider will need to monitor you closely for at least 16 weeks after you stop receiving it. Stopping Ultomiris may cause a breakdown of your red blood cells due to PNH. Symptoms or problems that can happen due to red blood cell breakdown include:

• drop in your red blood cell count
• shortness of breath
• tiredness
• blood clots
• blood in your urine
• trouble swallowing
• stomach-area (abdomen) pain
• erectile dysfunction (ED) in males.

If you have aHUS, your healthcare provider will need to monitor you closely for at least 12 months after stopping treatment for signs of worsening aHUS or problems related to a type of abnormal clotting and breakdown of your red blood cells called thrombotic microangiopathy (TMA). Symptoms or problems that can happen with TMA may include:

• confusion or loss of consciousness
• difficulty breathing
• seizures
• blood clots or stroke
• chest pain (angina).

What happens if I miss a dose?

If you miss an Ultomiris IV or SC  dose or administer a partial SC dose, call your healthcare provider right away.

What are the side effects of Ultomiris?

Ultomiris can cause the following serious side effects.

An increased risk of developing serious meningococcal infections caused by Neisseria meningitidis bacteria.

These may quickly become life-threatening or cause death if not recognized and treated early.

• You must complete or update your meningococcal vaccine(s) at least 2 weeks before your first dose.
• If you have not completed your meningococcal vaccines and Ultomiris must be started right away, you should receive the required vaccine(s) as soon as possible.
• If you have not been vaccinated and Ultomiris must be started right away, you should also receive antibiotics to take for as long as your healthcare provider tells you.
• If you had a meningococcal vaccine in the past, you might need additional vaccines before starting Ultomiris treatment. Your healthcare provider will decide if you need additional meningococcal vaccines.
• Meningococcal vaccines do not prevent all meningococcal infections. Call your healthcare provider or get emergency medical care right away if you get any of these signs and symptoms of a serious meningococcal infection:
  • fever with or without a rash
  • fever with a high heart rate
  • headache with nausea or vomiting
  • headache and fever
  • headache with a stiff neck or stiff back
  • confusion
  • eyes sensitive to light
  • muscle aches with flu-like symptoms.
• Your healthcare provider will give you a Patient Safety Card about the risk of serious meningococcal infection. Carry it with you at all times during treatment and for 8 months after your last dose of Ultomiris. Your risk of meningococcal infection may continue for several months after your last dose. It is important to show this card to any healthcare provider who treats you. This will help them diagnose and treat you quickly.

Other serious infections

Other types of serious infections caused by encapsulated bacteria, including Streptococcus pneumoniae, Haemophilus influenzae, and Neisseria gonorrhoeae.

• If your child is treated with Ultomiris, your child should receive vaccines against S. pneumonia and H. influenzae type b (Hib).

Gonorrhea

Certain people may be at risk of serious infections with gonorrhea. Talk to your healthcare provider about whether you are at risk for gonorrhea infection, about gonorrhea prevention, and regular testing.

Infusion-related reactions

These may happen during your IV or SC treatment.  Symptoms of an infusion-related reaction may include lower back pain, abdominal pain, muscle spasms, changes in blood pressure, tiredness, feeling faint, shaking chills (rigors), discomfort in your arms or legs, bad taste, or drowsiness. Stop Ultomiris and tell your healthcare provider right away if you develop these symptoms, or any other symptoms during your infusion that may mean you are having a serious infusion-related reaction, including:

• chest pain
• swelling of your face, tongue, or throat
• trouble breathing, or shortness of breath
• feeling faint or like you might pass out.

Allergic reactions to the acrylic adhesive used on the on-body injector

If you have an allergic reaction during the delivery of SC Ultomiris, remove the on-body injector and get medical help right away. Your healthcare provider may treat you with medicines to help prevent or treat allergic reaction symptoms as needed.

The most common side effects of Ultomiris reported, regardless of the condition being treated, include:

• upper respiratory tract infections, including COVID-19 infections
• headache
• local injection site reactions
• diarrhea
• high blood pressure
• nausea
• fever
• vomiting
• urinary tract infections
• joint pain (arthralgia)
• back pain.

Report suspected side effects to Alexion Pharmaceuticals, Inc. at 1-844-259-6783 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

What other drugs will affect Ultomiris?

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Ultomiris and other medicines can affect each other causing side effects.

• A supplemental dose of Ultomiris is required for patients administered intravenous immunoglobulins or undergoing plasma exchange, plasmapheresis
• Monitor patients taking neonatal Fc Receptor Blockers (FcRn), such as efgartigimod or rozanolixizumab, closely for reduced effectiveness of Ultomiris.

Know the medicines you take and the vaccines you receive. Keep a list of them to show your healthcare provider and pharmacist when you get a new medicine. See the prescribing information for a full list of interactions.

Storage

Ultomiris vials for intravenous infusion

Store refrigerated at 2°C - 8°C (36°F - 46°F) in the original carton to protect from light. Do not freeze. Do not shake. The vial requires dilution (refer to the product information).

If the infusion is not used immediately, store in the refrigerator at 2°C - 8°C (36°F - 46°F) for no longer than 24 hours taking into account the expected infusion time.

Ultomiris prefilled cartridges and on-body injector for subcutaneous administration

Store prefilled cartridges and on-body injectors refrigerated at 2°C - 8°C (36°F - 46°F) in the original carton to protect them from light and physical damage. Do not freeze. Do not shake or drop.

• Do not allow the on-body injector to get wet from water or other liquids.
• If removed from the refrigerator, store the cartridges and on-body injector in the original carton box at room temperature between 68°F - 77°F (20°C -25°C) for up to 3 days.
• Discard after 3 days if not used. Do not return to the refrigerator.

The single-use on-body injector with a prefilled cartridge is not made with natural rubber latex.

Ingredients

Active ingredient: ravulizumab-cwvz.

Inactive ingredients (intravenous injection):

• 100 mg/mL: L-arginine, polysorbate 80 (vegetable origin), sodium phosphate dibasic, sodium phosphate, monobasic, sucrose, and Water for Injection.
• 10 mg/mL: polysorbate 80 (vegetable origin), sodium chloride, sodium phosphate dibasic, sodium phosphate monobasic, and Water for Injection.

Inactive ingredients (subcutaneous injection):

• 70 mg/mL: L-arginine, polysorbate 80 (vegetable origin), sodium phosphate dibasic, sodium phosphate monobasic, sucrose, and Water for Injection.

Manufacturer

Alexion Pharmaceuticals, Inc.

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