Ultomiris (Alexion Pharma, 300 mg, 1100 mg): Uses, Side Effects and Dosage

Serious warnings and precautions box

Consult your doctor before you take Ultomiris to be sure that you receive vaccination against Neisseria meningitidis prior to, or at the time of, beginning therapy. If you start therapy less than 2 weeks after being vaccinated, you must take antibiotics to reduce the risk of infection for 2 weeks. Ensure that your current meningococcal vaccinations are up to date. You should also be aware that vaccination may not prevent this type of infection. In accordance with national recommendations, your doctor might consider that you need supplementary measures to prevent infection.

Meningococcal infection symptoms

Because of the importance of rapidly identifying and treating meningococcal infection in patients who receive Ultomiris, you will be provided a card to carry with you at all times, listing relevant signs and symptoms of meningococcal infection/sepsis. This card is named: “Patient Safety Card”.

If you experience any of the following symptoms, you should immediately inform your doctor:

• headache with nausea or vomiting
• headache and a fever
• headache with a stiff neck or stiff back
• fever
• fever and rash
• confusion
• muscle aches with flu-like symptoms
• eyes sensitive to light

What ULTOMIRIS is used for:

Paroxysmal Nocturnal Hemoglobinuria

Ultomiris is used for the treatment of adult and pediatric patients one month of age and older with a certain type of disease affecting the blood system called Paroxysmal Nocturnal Hemoglobinuria (PNH).

In patients with PNH, their red blood cells can be destroyed which can lead to low blood counts (anemia), tiredness, difficulty in functioning, pain, dark urine, shortness of breath, and blood clots.

atypical Hemolytic Uremic Syndrome

Ultomiris is used for the treatment of adult and pediatric patients one month of age and older with atypical Hemolytic Uremic Syndrome (aHUS).

In patients with aHUS, their kidneys and blood vessels, including platelets, can be inflamed which can lead to low blood counts (thrombocytopenia and anemia), reduced or lost kidney function, blood clots, tiredness and difficulty in functioning.

Generalized Myasthenia Gravis

Ultomiris is used to treat adult patients with a certain type of disease affecting the muscles called generalized Myasthenia Gravis (gMG).

In patients with gMG, their muscles can be damaged by their immune system, which can cause severe muscle weakness, impaired vision and mobility, shortness of breath, extreme fatigue, risk for aspiration (i.e. choking caused by inhaling food or liquid), and decreased ability to perform activities of daily living.

Neuromyelitis Optica Spectrum Disorder

Ultomiris is used to treat adult patients with a disease of the central nervous system that mainly affects the eye nerves and the spinal cord called neuromyelitis optica spectrum disorder (NMOSD).

In patients with NMOSD, the eye nerves and spinal cord are attacked and damaged by

the immune system.

How ULTOMIRIS works:

Ultomiris contains the active substance ravulizumab and that belongs to a class of medicines called monoclonal antibodies. Ravulizumab attaches to and stops the action of a specific protein in the body preventing one very specific component of your immune system from attacking and destroying vulnerable and needed blood, tissue and muscle cells.

In patients with NMOSD, the eye nerves and spinal cord are attacked and damaged by the immune system. Ultomiris is presumed to block a specific area of the body’s inflammatory response, and its ability to attack and destroy its own eye nerves and spinal cord.

The ingredients in ULTOMIRIS are:

Medicinal ingredients: Ravulizumab

100 mg/mL

Non-medicinal ingredients: L-arginine, polysorbate 80 (vegetable origin), sodium phosphate dibasic, sodium phosphate monobasic, sucrose, water for injection.

10 mg/mL

Non-medicinal ingredients: Polysorbate 80 (vegetable origin), sodium chloride, sodium phosphate dibasic, sodium phosphate monobasic, water for injection.

ULTOMIRIS comes in the following dosage forms:

100 mg/mL

Single use vial of 300 mg concentrate for solution for infusion; 3 mL

Single use vial of 1,100 mg concentrate for solution for infusion; 11 mL

10 mg/mL

Single use vial of 300 mg concentrate for solution for infusion; 30 mL

Do not use ULTOMIRIS if:

• you have not received a meningococcal vaccine before or at the time of starting therapy

To help avoid side effects and ensure proper use, talk to your healthcare professional before you take ULTOMIRIS. Talk about any health conditions or problems you may have, including if you:

• have an infection (including if you have or are at risk for gonorrhea infection)
• have any other unresolved infections

Other warnings you should know about:

Allergic Reactions

Ultomiris contains a protein than may cause allergic reactions in some people. Tell your doctor or nurse right away if you get any of these symptoms during your Ultomiris infusion:

• chest pain
• trouble breathing or shortness of breath
• swelling of your face, tongue, or throat
• feel faint or pass out

Infusion reactions

When Ultomiris is given, you may experience reactions to the infusion (drip) such as headache, lower back pain, and infusion-related pain. Some patients may experience infusion reactions and allergic or hypersensitivity reactions (including anaphylaxis, a serious allergic reaction which causes difficulty breathing or dizziness). Tell your doctor or nurse right away if you develop these symptoms, or any other symptoms during your Ultomiris infusion.

Pregnancy and Nursing

Ultomiris should not be used during pregnancy unless the potential benefit justifies the potential risk to the mother and the fetus.

The use of effective contraception during treatment and up to 8 months after treatment should be considered in women who are able to get pregnant.

If you are pregnant or breast-feeding, think you may be pregnant, or are planning to have a baby, ask your doctor or pharmacist for advice before using this medicine.

Ultomiris contains sodium

Ultomiris 10 mg/mL when diluted with 0.9% sodium chloride for IV administration contains 2.65 g sodium per 720 mL at the maximum dose, which is above Health Canada’s maximum daily intake recommendation of 2.3 g sodium. This should be taken into consideration by patients on a controlled sodium diet.

Ultomiris 100 mg/mL when diluted with 0.9% sodium chloride for IV administration contains 0.18 g sodium per 72 mL at the maximum dose, which is below Health Canada’s maximum daily intake recommendation of 2.3 g sodium.

Tell your healthcare professional about all the medicines you take, including any drugs, vitamins, minerals, natural supplements or alternative medicines.

How to take ULTOMIRIS:

The treatment will be given by your doctor or other health care provider by infusing a dilution of the Ultomiris vial from a drip bag through a tube directly into one of your veins. It is recommended that the beginning of your treatments, called the loading phase, will extend over 2 weeks, followed by a maintenance phase. The doses administered are based on your body weight, as shown in Table 1, your doctor will calculate this. Two weeks after receiving your loading dose, you will be administered Ultomiris once every 8 weeks.

If you were receiving Soliris prior to receiving Ultomiris, the loading dose should be administered 2 weeks after the last Soliris infusion. The infusion will take approximately 2 hours.

Pediatric patients:

Pediatric patients who weigh ≥ 40 kg are treated with the adult dosing recommendations. The weight-based dosing recommendation and dosing interval for pediatric patients below < 40 kg are shown in Table 1 with maintenance doses starting 2 weeks after loading dose administration.

Table 1: Ultomiris Weight-Based Dosing Regimen

Administration of PE/PI (plasmapheresis or plasma exchange, or fresh frozen plasma infusion) or intravenous immunoglobulin (IVIg) may reduce Ultomiris serum levels.

Overdose:

Missed dose:

If you forget an appointment, please contact your doctor immediately for advice, and see section below “If you stop using Ultomiris.”

If you stop using Ultomiris for:

PNH

Interrupting or ending treatment with Ultomiris may cause your PNH symptoms to return with greater severity. Your doctor will discuss the possible side effects with you and explain the risks. Your doctor will want to monitor you closely for at least 16 weeks.

The risks of stopping Ultomiris include an increase in the destruction of your red blood cells, which may cause:

• a significant fall in your red blood cell counts (anemia),
• confusion or change in how alert you are,
• chest pain, or angina,
• an increase in your serum creatinine level (problems with your kidneys), or
• thrombosis (blood clotting).

aHUS

If you have aHUS, your doctor 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
• seizures
• chest pain (angina)
• difficulty breathing
• blood clots or stroke

gMG

Interrupting or stopping treatment with Ultomiris may cause your gMG symptoms to reoccur or become worse. Talk to your doctor about the management of your gMG before stopping Ultomiris.

They will discuss with you the possible side effects and risks of interrupting or stopping treatment with Ultomiris.

NMOSD

Interrupting or ending treatment for Ultomiris may cause your NMOSD symptoms to reoccur or become worse. Talk to your doctor about the management of your NMOSD before stopping Ultomiris. They will discuss with you the possible side effects and risks of interrupting or stopping treatment with Ultomiris.

Symptoms of NMOSD may include:

• loss of sight in one or both eyes
• weakness or loss of movement in the legs or arms
• painful spasms
• loss of feeling
• problems with bladder and bowel function

If you have any of these symptoms, contact your doctor.

If you have any further questions on the use of this medicine, ask your doctor.

Possible side effects from using ULTOMIRIS:

These are not all the possible side effects you may have when taking ULTOMIRIS. If you experience any side effects not listed here, tell your healthcare professional.

The most serious side effect is meningococcal infection/sepsis.

If you experience any of the meningococcal infection symptoms, you should immediately inform your doctor. Symptoms are listed under the ‘Serious Warnings & Precautions’ Box and in your Patient Safety Card.

The most common side effects in adult patients with PNH treated with Ultomiris are upper respiratory infection and headache.

The most common side effects of Ultomiris in pediatric patients with PNH are abdominal pain and common cold.

The most common side effects of Ultomiris in patients with aHUS are upper respiratory tract infection, common cold and headache.

The most common side effects of Ultomiris in people with gMG are diarrhea and upper respiratory tract infection.

The most common side effects of Ultomiris in adult patients with NMOSD are headache, back pain, arthralgia, urinary tract infection, common cold, sinus infection, fever, diarrhea, feeling sick (nausea).,

Serious side effects and what to do about them

^a Estimated from postmarketing experience

^b Gonococcal infection includes disseminated gonococcal infection

^cHypersensitivity is a group term for Preferred Term drug hypersensitivity with related causality and Preferred Term hypersensitivity

If you have a troublesome symptom or side effect that is not listed here or becomes bad enough to interfere with your daily activities, tell your healthcare professional.

Reporting side effects

Storage:

Do not use this medicine after the expiry date which is stated on the carton after “EXP”. The expiry date refers to the last day of that month.

Store in a refrigerator (2 to 8°C).

Do not freeze. Do not shake.

Keep out of reach and sight of children.

Store in the original package in order to protect from light.

If you want more information about ULTOMIRIS:

• Talk to your healthcare professional
• Find the full product monograph that is prepared for healthcare professionals and includes the Patient Medication Information by visiting the Health Canada Drug Product Database website (https://www.canada.ca/en/health-canada/services/drugs-health-products/drug-products/drug-product-database.html); the manufacturer’s website www.alexion.com; or by calling 1-888-765-4747.

Date of Authorization: 2026-08-05