Ultomiris is a monoclonal antibody. Monoclonal antibodies are man-made antibodies that fight antigens (harmful substances) in the body.

Ultomiris injection is used to treat paroxysmal nocturnal hemoglobinuria (PNH) in adults and children who are at least 1 month old.

PNH is a rare genetic disorder in which defective red blood cells break down prematurely and leak hemoglobin into your blood. Hemoglobin is the iron-rich protein that gives blood its red color. When hemoglobin passes into your urine, it can appear dark or tea-colored (especially in the morning or after sleep, when the urine is most concentrated).

Ultomiris is used to treat a rare chronic blood disease called hemolytic uremic syndrome (aHUS) in adults and children who are at least 1 month old. This medicine is not for use in treating HUS that is related to Shiga toxin E. coli.

Ultomiris is also used to treat adults with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive. 

Ultomiris is available only under a special program. You must be registered in the program and understand the risks and benefits of this medicine.

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

You will need to receive certain vaccinations before you start using Ultomiris.

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.

Use Ultomiris exactly as prescribed by your doctor. Follow all directions on your prescription label and read all medication guides or instruction sheets. Your doctor may occasionally change your 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.

Ultomiris is injected into a vein by a healthcare provider. The first two infusions are usually given 2 weeks apart, followed by an infusion once every 4-8 weeks.

After each infusion, you will be watched closely for at least 1 hour to make sure you do not have an allergic reaction.

Doses are based on weight. Your dose may change if you gain or lose weight.

You may get infections more easily, even serious or fatal infections. You will need frequent medical tests.

Read the Patient Safety Card about serious infections and the symptoms to watch for. 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). Talk with your doctor about safe ways to keep from getting an infection during sex.

Ultomiris can have long lasting effects on your body, even after you stop using this medicine. If you have PNH and you stop using Ultomiris, your doctor may need to check your progress for at least 16 weeks after your last dose. If you have aHUS and you stop using Ultomiris, your doctor may need to check you for at least 12 months after your last dose.

Usual Adult and Pediatric Dose for Paroxysmal Nocturnal Hemoglobinuria:

5 kg to less than 10 kg:
-Loading dose: 600 mg IV
-Maintenance dose: 300 mg IV every 4 weeks starting 2 weeks after the loading dose
10 kg to less than 20 kg:
-Loading dose: 600 mg IV
-Maintenance dose: 600 mg IV every 4 weeks starting 2 weeks after the loading dose
20 kg to less than 30 kg:
-Loading dose: 900 mg IV
-Maintenance dose: 2100 mg IV every 8 weeks starting 2 weeks after the loading dose
30 kg to less than 40 kg:
-Loading dose: 1200 mg IV
-Maintenance dose: 2700 mg IV every 8 weeks starting 2 weeks after the loading dose
40 kg to less than 60 kg:
-Loading dose: 2400 mg IV
-Maintenance dose: 3000 mg IV every 8 weeks starting 2 weeks after the loading dose
60 kg to less than 100 kg:
-Loading dose: 2700 mg IV
-Maintenance dose: 3300 mg IV every 8 weeks starting 2 weeks after the loading dose
100 kg or greater:
-Loading dose: 3000 mg IV
-Maintenance dose: 3600 mg IV every 8 weeks starting 2 weeks after the loading dose.

Use: For the treatment of adults and pediatric patients one month and older with paroxysmal nocturnal hemoglobinuria (PNH)

Usual Adult and Pediatric Dose for Hemolytic Uremic Syndrome:

5 kg to less than 10 kg:
-Loading dose: 600 mg IV
-Maintenance dose: 300 mg IV every 4 weeks starting 2 weeks after the loading dose
10 kg to less than 20 kg:
-Loading dose: 600 mg IV
-Maintenance dose: 600 mg IV every 4 weeks starting 2 weeks after the loading dose
20 kg to less than 30 kg:
-Loading dose: 900 mg IV
-Maintenance dose: 2100 mg IV every 8 weeks starting 2 weeks after the loading dose
30 kg to less than 40 kg:
-Loading dose: 1200 mg IV
-Maintenance dose: 2700 mg IV every 8 weeks starting 2 weeks after the loading dose
40 kg to less than 60 kg:
-Loading dose: 2400 mg IV
-Maintenance dose: 3000 mg IV every 8 weeks starting 2 weeks after the loading dose
60 kg to less than 100 kg:
-Loading dose: 2700 mg IV
-Maintenance dose: 3300 mg IV every 8 weeks starting 2 weeks after the loading dose
100 kg or greater:
-Loading dose: 3000 mg IV
-Maintenance dose: 3600 mg IV every 8 weeks starting 2 weeks after the loading dose.

Use: For the treatment of adults and pediatric patients one month and older with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA)

Usual Adult Dose for Myasthenia Gravis

40 kg to less than 60 kg:
-Loading dose: 2400 mg IV
-Maintenance dose: 3000 mg IV every 8 weeks starting 2 weeks after the loading dose
60 kg to less than 100 kg:
-Loading dose: 2700 mg IV
-Maintenance dose: 3300 mg IV every 8 weeks starting 2 weeks after the loading dose
100 kg or greater:
-Loading dose: 3000 mg IV
-Maintenance dose: 3600 mg IV every 8 weeks starting 2 weeks after the loading dose.

Use: For the treatment of adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive

You should not be treated with Ultomiris if you are allergic to ravulizumab, if you 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.

Tell your doctor if you have recently had any symptoms of infection (fever, chills, or flu-like symptoms).

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.

Do not breastfeed while using Ultomiris, and for at least 8 months after your last dose.

Ultomiris is not approved for use by anyone younger than 1 month old.

Call your doctor for instructions if you miss an appointment for your Ultomiris injection.

In a medical setting an overdose would be treated quickly.

Follow your doctor's instructions about any restrictions on food, beverages, or activity.

Get emergency medical help if you have signs of an allergic reaction to Ultomiris: hives; difficult breathing; swelling of your face, lips, tongue, or throat.

Some side effects may occur during the injection. Tell your caregiver if you feel light-headed or if you have chest pain, trouble breathing, or swelling in your face.

Call your doctor right away if you have signs of infection such as:

• muscle pain with flu-like symptoms;

• fever and a rash;

• fever and a headache;

• headache and stiffness in your neck or back;

• headache and nausea or vomiting;

• confusion; or

• your eyes may be more sensitive to light.

Call your doctor at once if you have symptoms of gonorrhea, such as:

• pain or burning when you urinate;

• pain or swelling of the genital or rectal area;

• unusual vaginal bleeding; or

• foul discharge from the penis or vagina.

If you stop using Ultomiris, tell your doctor if you have any new or worsening symptoms, such as: tiredness, confusion, stomach pain, chest pain, trouble breathing or swallowing, (in men) trouble having an erection, blood in your urine, a seizure, or loss of consciousness.

Common Ultomiris side effects may include:

• fever;

• high blood pressure;

• diarrhea, nausea, vomiting;

• headache; or

• cold symptoms such as stuffy nose, sneezing, sore throat.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Other drugs may interact with ravulizumab, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all other medicines you use.