Key takeaways

  • Ultomiris is a biologic medication administered through intravenous infusion. It works by blocking a specific protein in the immune system to treat four rare autoimmune conditions: paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, generalized myasthenia gravis, and neuromyelitis optica spectrum disorder.
  • Ultomiris carries a significant risk of life-threatening meningococcal infections, requiring patients to receive meningococcal vaccines at least two weeks before starting treatment and to watch for warning signs like sudden high fever, severe headache, stiff neck, or confusion.
  • Treatment with Ultomiris typically involves a higher initial loading dose followed by regular maintenance infusions every four to eight weeks, with dosing based on body weight and the specific condition being treated.

Ultomiris (ravulizumab-cwvz) is a prescription drug approved to treat certain rare inflammatory conditions. Ultomiris comes as a liquid solution for intravenous (IV) infusion, which is given by a healthcare professional.

Specifically, Ultomiris is used to treat:

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

Ultomiris basics

Ultomiris contains the active ingredient ravulizumab-cwvz.

Ultomiris is a biologic drug, which means it’s made from parts of living organisms. It’s available only as a brand-name medication. It isn’t available in a biosimilar form. Biosimilars are like generic drugs. Just as generics can be an alternative to brand-name medications, biosimilars are an effective alternative to biologics.

Biosimilars will have the same active ingredient core name as the brand-name drug, but with unique suffixes. This is why “-cwvz” appears at the end of the name of the active ingredient — to show it’s distinct from biosimilars that may be created in the future.

Ultomiris has a boxed warning. This is the most serious warning from the Food and Drug Administration (FDA). For details, see the “Ultomiris precautions” section.

Ultomiris is FDA approved to treat multiple conditions in certain adults and children. It belongs to a group of drugs called complement inhibitors. (Ultomiris is not chemotherapy.) Its mechanism of action (how it works) is by blocking the action of a specific protein called C5 in your body’s complement system. By blocking the C5 action, Ultomiris stops activity in the complement system that’s causing the condition being treated.

Keep reading to learn more about each of the conditions Ultomiris treats and how specifically the drug works to treat them.

Ultomiris for paroxysmal nocturnal hemoglobinuria (PNH)

Ultomiris is used to treat PNH, a rare autoimmune and blood disorder. PNH leads to the destruction of irregular red blood cells through a process called hemolysis. This causes hemoglobin to be released into the urine, resulting in red or dark-colored urine. Hemolysis can occur constantly but may be more frequent at night or during times of immune system activation.

Common symptoms of PNH include fatigue, weakness, dizziness, fast heart rate, shortness of breath, headache, and chest pain.

Severe cases may require blood transfusions and can lead to complications such as difficulty swallowing, abdominal spasms, and erectile dysfunction. PNH can also lead to blood clotting issues, potentially causing blockages in vital organs like the brain, liver, stomach, or kidneys.

For people with PNH, Ultomiris helps prevent the complement system from destroying the red blood cells. It helps stabilize your hemoglobin levels and reduces symptoms of anemia. It also decreases your risk of blood clots and your need for blood transfusions.

Ultomiris for atypical hemolytic uremic syndrome (aHUS)

Ultomiris is used to treat aHUS. This is a long-term autoimmune condition triggered by factors such as infection, pregnancy, cancer, injury, or surgery.

With aHUS, the immune system damages small blood vessels. This leads to blood clot formation that can block blood flow to vital organs — particularly the kidneys, brain, heart, or digestive tract — and cause organ damage.

Symptoms of aHUS can appear slowly or suddenly and may include easy bruising, skin discoloration, headaches, fatigue, breathing difficulties, blood in urine or stool, dark urine, reduced urination, swelling, nausea, vomiting, confusion, and high blood pressure.

Note: Ultomiris is not approved to treat Shiga toxin E. coli-related hemolytic uremic syndrome (STEC-HUS), a more common form of HUS known as “typical HUS.”

In people with aHUS, Ultomiris helps prevent the complement system from damaging your small blood vessels. This can help stop blood clots from forming in these vessels, which helps prevent organ damage. It also allows your platelet and red blood cell levels to return to usual.

Ultomiris for generalized myasthenia gravis (gMG)

Ultomiris is used to treat gMG in people with anti-acetylcholine receptor (AChR) antibodies.

With gMG, your immune system produces antibodies that attack muscle receptor cells. This prevents your nerves from sending the signals to your muscles and your muscles from receiving the signals they need in order to move. This can cause symptoms such as muscle weakness and fatigue.

For people with gMG, Ultomiris helps prevent the complement system from attacking nerve receptors in your muscles. This helps nerve signals reach the muscles, which improves their strength and lessens fatigue.

Ultomiris for neuromyelitis optica spectrum disorder (NMOSD)

Ultomiris is used to treat NMOSD, which is also called neuromyelitis optica or Devic’s disease. With this condition, your immune system damages cells in your eyes and spinal cord.

For people with NMOSD, Ultomiris helps stop the complement system from attacking your central nervous system, especially your optic nerves and spinal cord. This reduces inflammation and damage to these areas. It also helps prevent vision problems and other symptoms of NMOSD.

Like most drugs, Ultomiris may cause mild to serious side effects.

The most common side effects of Ultomiris reported in studies of the drug varied based on the condition it was used to treat and included:

Ultomiris also has a boxed warning about the risk of serious meningococcal infections (see the “Ultomiris precautions” section for details).

Ultomiris doses are given via intravenous (IV) infusions, usually at a doctor’s office or clinic every few weeks.

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

  • the type and severity of the condition you’re being prescribed Ultomiris to treat
  • your age and body weight
  • other medical conditions you may have

Also, your doctor may start your Ultomiris treatment with a loading dose. (A loading dose is a higher dose of medication given at the beginning of treatment to help the drug start working quickly.)

Ultomiris is not known to interact with other medications, herbs, vitamins, supplements, or foods. The drug’s manufacturer didn’t look at interactions in studies. But this doesn’t mean that interactions can’t happen with Ultomiris.

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

If you have questions about Ultomiris interactions that may affect you, talk with your doctor or pharmacist.

Ultomiris and alcohol

Alcohol is not known to interact with Ultomiris. However, if you have certain side effects with Ultomiris, such as headaches or dizziness, drinking alcohol could make them worse. Because of this, your doctor may recommend that you limit the amount of alcohol you drink.

Talk with your doctor to determine whether it’s safe for you to consume alcohol during Ultomiris treatment.

Whether you have health insurance or not, cost may be a factor when you’re considering Ultomiris. What you’ll pay for this medication may depend on several things, such as your insurance plan, your location, and where you receive your infusions.

Here are a few things to consider regarding cost:

  • Cost information and savings coupons: You can visit Optum Perks for price estimates of Ultomiris. These estimates are based on the use of Optum Perks coupons, which cannot be used with insurance copays or benefits.
  • Savings program: Alexion, the manufacturer of Ultomiris, offers a program called OneSource that provides financial assistance and other kinds of support for people who qualify. For more information and to find out if you’re eligible, call 888-765-4747 or visit the program website.

Talk with your doctor or pharmacist if you have questions about how to pay for Ultomiris.

Optum Perks is owned by RVO Health. By clicking on this link, we may receive a commission. Learn more.

The following is important information to consider and discuss with your doctor before starting Ultomiris treatment.

Pregnancy and Ultomiris

It’s not known whether Ultomiris is safe to receive during pregnancy, as this medication hasn’t been studied in pregnant people.

In animal studies, a drug similar to Ultomiris called eculizumab (Soliris) caused problems with fetal development (commonly known as birth defects) in pregnant females. But animal studies do not always predict what will happen in humans.

It’s important to note that if you’re pregnant and have one of the conditions Ultomiris is approved to treat, your condition has serious risks. Some of these risks can affect both you and your child. They include blood clots, bleeding, pregnancy loss, and premature delivery.

If you’re planning a pregnancy or can become pregnant, talk with your doctor about potential treatments for your condition. Together, you can consider the risks and benefits of your treatment options and decide whether you should receive Ultomiris infusions during this time.

Birth control and Ultomiris

It’s not known whether Ultomiris treatment is safe during pregnancy. If you’re sexually active and you or your partner can become pregnant, talk with your doctor about your birth control options during Ultomiris treatment.

Ultomiris and breastfeeding

It’s unknown whether Ultomiris passes into breast milk, but if it does, it could cause serious side effects in a child who’s breastfed. Because of this, your doctor will likely advise against breastfeeding during Ultomiris treatment and for 8 months following your last dose.

If you’re breastfeeding or plan to breastfeed, talk with your doctor about other healthy ways to feed your child during your Ultomiris treatment.

This section describes the warnings and precautions for Ultomiris.

FDA warning: Serious meningococcal infections

Ultomiris has a boxed warning about the risk of serious meningococcal infections. A boxed warning is the most serious warning from the Food and Drug Administration (FDA). It alerts doctors and patients about drug effects that may be dangerous.

Ultomiris treatment increases the risk of serious infections such as bacterial meningitis and sepsis. These infections can be life threatening unless treated quickly. If you’re concerned about your risk of infection with Ultomiris, talk with your doctor before starting treatment.

Because of this risk, you’ll receive meningococcal vaccines at least 2 weeks before your first Ultomiris infusion. These vaccines reduce your risk of contracting meningococcal infections, but you may still get sick.

See your doctor right away or go to the nearest emergency room if you have:

To help reduce the risk of infection, Ultomiris is only available through a restricted risk evaluation and mitigation strategy (REMS) program. If you have questions about the REMS program or the risk of meningococcal infection with Ultomiris, visit the program website or talk with your doctor.

Other precautions

Before starting Ultomiris, discuss your health history with your doctor. This medication 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:

  • current or recurring infections
  • previous allergic reaction to this or a similar drug
  • pregnancy
  • breastfeeding

Ultomiris (ravulizumab-cwvz) is a long-lasting prescription medicine used to treat several rare diseases where the body’s immune system mistakenly attacks its own healthy cells. It works by blocking a specific protein in the blood to prevent it from causing inflammation and damage to organs like the kidneys, eyes, or muscles.

Ultomiris is administered via an IV infusion, usually at a doctor’s office or clinic every few weeks. This drug carries a risk of severe infections, so doctors must monitor patients closely and ensure they are up to date on their vaccines. More common side effects of Ultomiris include headaches, diarrhea, and fatigue.

Disclaimer: While Healthline strives for factual, comprehensive, and current information, this article is not a substitute for a licensed healthcare professional’s expertise. Always consult a doctor before taking or discontinuing any medication. Drug information is subject to change and may not cover all uses, directions, precautions, warnings, interactions, reactions, or adverse effects. The lack of a warning does not guarantee a drug’s safety, effectiveness, or appropriateness for all patients or specific uses. Learn more about our approach to content integrity.