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:
- generalized myasthenia gravis (gMG) in adults who test positive for anti-acetylcholine receptor antibodies
- atypical hemolytic uremic syndrome (aHUS) in adults and some children
- paroxysmal nocturnal hemoglobinuria (PNH) in adults and some children
- neuromyelitis optica spectrum disorder (NMOSD) in certain adults
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 “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 following lists contain some of the more common side effects this drug 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 Ultomiris. They can also suggest ways to help reduce or manage them.
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 Ultomiris 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 Ultomiris can cause. To learn about other mild side effects, talk with your doctor or pharmacist, or read the drug’s prescribing information.
Mild side effects of Ultomiris that have been reported include:
- abdominal pain
- anxiety
- constipation
- diarrhea
- dizziness
- fatigue
- fever
- headache
- low red blood cell levels
- muscle spasms
- nausea, vomiting
- pain in the arms, legs, back, muscles, or joints
- shortness of breath
- skin rash
- swelling in the arms and legs
- upper respiratory tract infection
- urinary tract infection (UTI)
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 Ultomiris can occur, but they aren’t common. If you have serious side effects from this medication, call your doctor right away. If you think you’re having a medical emergency, call 911 or your local emergency number.
Serious side effects of Ultomiris that have been reported include:
- boxed warning: risk of serious meningococcal infection
- high blood pressure
- hyperthermia, a condition in which the body’s temperature is too high
- infusion-related reactions, such as low back pain and blood pressure changes
- serious infection, such as pneumonia or tonsillitis
For more information about side effects related to boxed warnings, see the “Precautions” section.
Side effects in children
Ultomiris is approved to treat paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) in children ages 1 month and older.
In the drug’s studies, children and adults experienced similar side effects. The most common side effects in children were:
- abdominal pain
- headache
- low levels of red blood cells
- upper respiratory tract infections
Children also have a risk of meningococcal infection from Ultomiris. Babies can experience different symptoms from this condition compared with adults and older children. These include:
- being slow or inactive
- developing a bulge in the soft spot on their skull
- irritability
- lacking appetite
- vomiting
If your baby has any of these symptoms during Ultomiris treatment, contact their doctor right away.
Talk with your child’s doctor or a pharmacist for more information about side effects that children of different ages may experience with Ultomiris.
Allergic reactionFor some people, Ultomiris can cause an allergic reaction, but these reactions were rare in the drug’s studies.
In general, symptoms of an allergic reaction can be mild or serious.
What might helpIf 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 Ultomiris 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 Ultomiris, they may have you switch to a different treatment.
The Ultomiris dosage your doctor prescribes will depend on several factors. These include:
- the type and severity of the condition you’re using Ultomiris to treat
- your age
- other medical conditions you may have
The following information describes dosages that are commonly used or recommended. But be sure to take the dosage your doctor prescribes for you. They’ll determine the best dosage to fit your needs.
Form and strengths
Ultomiris comes as a liquid solution that’s given as an intravenous (IV) infusion (an injection into a vein over a period of time). It comes in three strengths:
- 300 milligrams in 30 milliliters of liquid solution (10 mg/mL)
- 300 mg in 3 mL (100 mg/mL)
- 1,100 mg in 11 mL (100 mg/mL)
Recommended dosages
Your doctor will prescribe the dosage of Ultomiris that’s right for you. Keep reading to learn more.
Ultomiris dosage for PNH and aHUS
You’ll start treatment for paroxysmal nocturnal hemoglobinuria (PNH) or atypical hemolytic uremic syndrome (aHUS) by receiving one loading dose. (A loading dose is a higher dose of medication given at the beginning of treatment to help the drug start working quickly.)
Two weeks later, you’ll receive your first ongoing (maintenance) dose. Then, you’ll receive your next maintenance dose 4 or 8 weeks later. The following chart shows the typical dosing schedule for PNH and aHUS.
Note: Ultomiris dosing is based on your body weight in kilograms (kg). (One kg equals approximately 2.2 pounds [lb].)
| Weight in kg | Loading dose | Maintenance dose | Frequency |
|---|---|---|---|
| 5 kg to less than 10 kg | 600 mg | 300 mg | once every 4 weeks |
| 10 kg to less than 20 kg | 600 mg | 600 mg | once every 4 weeks |
| 20 kg to less than 30 kg | 900 mg | 2,100 mg | once every 8 weeks |
| 30 kg to less than 40 kg | 1,200 mg | 2,700 mg | once every 8 weeks |
| 40 kg to less than 60 kg | 2,400 mg | 3,000 mg | once every 8 weeks |
| 60 kg to less than 100 kg | 2,700 mg | 3,300 mg | once every 8 weeks |
| 100 kg or greater | 3,000 mg | 3,600 mg | once every 8 weeks |
Ultomiris dosage for gMG and NMOSD
You’ll start treatment for generalized myasthenia gravis (gMG) or neuromyelitis optica spectrum disorder (NMOSD) with one loading dose. (A loading dose is a higher dose of medication given at the beginning of treatment to help the drug start working quickly.)
You’ll receive your first maintenance (ongoing) dose 2 weeks later. Then, you’ll receive your next maintenance dose 8 weeks later. Your Ultomiris infusions for gMG will continue once every 8 weeks. See the following chart for the typical dosing schedule for gMG and NMOSD.
Note: Ultomiris dosing is based on your body weight in kg. (One kg equals approximately 2.2 lb.)
| Weight in kg | Loading dose | Maintenance dose | Dosing schedule |
|---|---|---|---|
| 40 kg to less than 60 kg | 2,400 mg | 3,000 mg | once every 8 weeks |
| 60 kg to less than 100 kg | 2,700 mg | 3,300 mg | once every 8 weeks |
| 100 kg or greater | 3,000 mg | 3,600 mg | once every 8 weeks |
Children’s dosage
Ultomiris is used to treat PNH and aHUS in children ages 1 month and older. It’s not approved to treat gMG or NMOSD in children.
Your child’s dosage is based on their body weight. For details, see the “Ultomiris dosage for PNH and aHUS” section.
If you have questions about your child’s dosage of Ultomiris, talk with their doctor.
How to use
Your doctor will explain how Ultomiris will be given to you. They’ll also explain how much you’ll be given and how often.
Ultomiris is available as a liquid solution that’s given as an intravenous (IV) infusion (an injection into your vein over a period of time). You’ll receive these infusions at a clinic or your doctor’s office.
The length of your infusion may depend on your specific dosage. It could take from 24 minutes to 90 minutes or longer.
For more information about Ultomiris treatment, talk with your doctor or visit the drug manufacturer’s website.
About receiving Ultomiris
The following are some common questions about receiving Ultomiris.
What if I miss a dose of Ultomiris?
If you miss an Ultomiris infusion appointment, call your doctor’s office as soon as possible. You should get your missed infusion within 7 days of when it was scheduled. You’ll then follow your original dosing schedule for the next dose.
If you miss an appointment and don’t get your missed dose within 7 days of when it was due, your doctor will adjust your future dosing schedule as needed.
If you need help remembering your infusion appointments, try using a medication reminder, such as a reminder app downloaded to your phone.
Will I need to use Ultomiris long term?
Yes, Ultomiris is typically a long-term treatment. If you and your doctor determine that it’s safe and effective for your condition, you’ll likely receive it long term.
How long does Ultomiris take to work?
Ultomiris begins to work right after you receive your first infusion, but it may take a few weeks before your symptoms start to improve.
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:
- confusion
- fever — with or without headache or skin rash
- flu-like symptoms with muscle aches
- light sensitivity
- nausea or vomiting with a headache
- stiff neck or back with a headache
To help reduce the risk of infection, Ultomiris is only available through a restricted
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
Note: For more information about the potential negative effects of Ultomiris, see the “Side effects of Ultomiris” section.
Other drugs are available that can treat your condition. If you’d like to explore an alternative to Ultomiris, talk with your doctor. They can tell you about other medications that might work well for you.
The following drugs are similar to Ultomiris:
Note: Some of the drugs listed here are used off-label for certain conditions Ultomiris is approved to treat. (With off-label use, a drug is prescribed for a condition it isn’t FDA approved to treat.)
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.