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Getting C5 under control is possible with ULTOMIRIS

ULTOMIRIS is a treatment that is designed to target the cause of PNH damage.

Why ULTOMIRIS?

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The underlying cause of PNH is ongoing hemolysis, and ULTOMIRIS targets complement, a key driver of hemolysis in PNH. ULTOMIRIS has been studied in the largest PNH trial to date and offers extended control of your PNH symptoms between infusions.

People taking ULTOMIRIS:
  • Had levels of LDH (a marker that measures PNH activity) that stayed stable over time, as did people taking SOLIRIS® (eculizumab)
  • Had few breakthrough events
    • Breakthrough events are defined as experiencing at least 1 new or worsening sign or symptom of hemolysis (eg, fatigue, hemoglobinuria, abdominal pain, shortness of breath, anemia).
    • In a clinical trial of people who had no prior PNH treatment, 4% of participants in the ULTOMIRIS group experienced breakthrough hemolysis vs 10.7% in the SOLIRIS group.
    • In a clinical trial of people who had prior PNH treatment, no one in the ULTOMIRIS group experienced breakthrough hemolysis vs 5.1% in the SOLIRIS group.

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See PNH and PNH treatment from an expert’s point of view

See PNH and PNH treatment from an expert’s point of view

In her video, Dr Anita Hill takes a closer look at what causes PNH disease activity and symptoms and how ULTOMIRIS can help.

ULTOMIRIS is designed to control intravascular hemolysis and PNH symptoms—with fewer infusions

Removes C5

ULTOMIRIS binds to the complement protein C5 to block its activation and
remove it from the bloodstream.

Reduces hemolysis

By binding and eliminating C5, ULTOMIRIS controls intravascular hemolysis (IVH)
of PNH red blood cells.

Long-acting
control

ULTOMIRIS stays in the body for a long period of time, allowing up to 8 weeks between infusions.a With the possibility of every-8-week dosing, ULTOMIRIS means your plans don’t have to center around frequent infusions.

ULTOMIRIS is the first and only FDA-approved, long-acting medication for adults and children 1 month of age and older with PNH.

Long-acting control of IVH means more time between infusions.

aTwo weeks after the intravenous loading dose, ULTOMIRIS is infused intravenously every 8 weeks for most people and every 4 weeks for children weighing less than 44 pounds (20 kilograms).

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Your healthcare provider will consider all test results, signs, and symptoms

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How can I help my healthcare provider monitor my PNH?

Track your signs, symptoms, and lab results. They will show you and your healthcare provider how you are physically affected by PNH.

Be sure to keep track of changes in your symptoms. Monitoring your symptoms is important, since PNH can manifest in serious ways. It can cause blood clots, which block veins and arteries and can lead to heart attack, stroke, and damage to your organs, as well as other problems.

Stay in touch with your healthcare provider about your PNH. Make sure you are on the same page and getting the most out of your treatment experience.

You don’t have to accept feeling sick

When you deal with PNH every day, over time, you may learn to cope with your symptoms. But it doesn’t have to be that way. You don’t have to accept feeling sick. Your healthcare provider can seek a treatment that is appropriate for you. That is why it is important to track your signs and symptoms: so you can tell if they’re getting worse over time instead of just accepting them. Talk to your healthcare provider about treatment options—you shouldn’t have to feel like feeling sick is normal.

Speaking with your healthcare provider is key to successful management of PNH

Lab tests that your healthcare provider may order

High-sensitivity
flow cytometry
to measure
clone size

  • Clone size is the percentage of blood cells in your body affected by PNH and, therefore, lack the protective proteins that blood cells usually have on their surface

  • This test measures the actual number of red and white blood cells affected by PNH in a small sample of circulating blood taken from your arm

  • It is the standard test for confirming whether or not you have PNH, and, through continued monitoring, your healthcare provider can tell if your clone size is changing over time

LDH level
to measure
hemolysis

  • Lactate dehydrogenase (LDH) is an enzyme found in red blood cells that is released during hemolysis

  • Knowing how much LDH is in your blood helps show how much hemolysis is happening in your body

  • Your LDH level, in comparison with your LDH level before starting ULTOMIRIS, may show how well you are responding to ULTOMIRIS

Hemoglobin
to test for
anemia

  • Hemoglobin is usually located inside red blood cells, but it is released into the bloodstream when the cells are destroyed by hemolysis. This form of hemoglobin, called serum free hemoglobin, is harmful and is the cause of many signs, symptoms, and serious health problems associated with PNH

  • The hemoglobin test that your healthcare provider might run to see if you have anemia measures the hemoglobin that is still located inside intact red blood cells, not serum free hemoglobin

  • Low hemoglobin levels in PNH patients may be due to PNH-related hemolysis, or they may be due to problems with red blood cell production caused by underlying bone marrow problems like aplastic anemia or myelodysplastic syndrome (MDS)

Platelet counts
to measure
platelet levels

  • The platelet count measures the amount of platelets in your blood. Platelets are involved in blood clot formation and play an important role in helping you heal from injury

  • PNH may also affect your platelet level

  • Your platelet count might stay the same even after months of treatment, regardless of a decrease in the LDH level and a need for blood transfusions

Creatinine
to assess
signs of
kidney damage

  • Levels of creatinine, a waste product in the blood, can show how well your kidneys are working

  • The creatinine level can help indicate if and how PNH is affecting your kidneys

ULTOMIRIS is designed to provide C5 control for up to 8 weeksb in people with PNH

ULTOMIRIS was studied in the largest clinical trial program of people with PNH to date.

SEE THE DATA

bTwo weeks after the intravenous loading dose, ULTOMIRIS is infused intravenously every 8 weeks for most people and every 4 weeks for children weighing less than 44 pounds (20 kilograms).

Understanding
ULTOMIRIS

ULTOMIRIS has an established safety profile in adults and children 1 month of age and older with PNH.

LEARN ABOUT ULTOMIRIS SAFETY

IMPORTANT SAFETY INFORMATION INCLUDING BOXED WARNING

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What is the most important information I should know about ULTOMIRIS?

ULTOMIRIS is a medicine that affects your immune system and may lower the ability of your immune system to fight infections.

  • ULTOMIRIS increases your chance of getting serious meningococcal infections that may quickly become life-threatening or cause death if not recognized and treated early.
  1. You must complete or update meningococcal vaccine(s) at least 2 weeks before your first dose of ULTOMIRIS.
  2. 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.
  3. If you have not been vaccinated and ULTOMIRIS must be started right away, you should also receive antibiotics for as long as your healthcare provider tells you.
  4. If you had a meningococcal vaccine in the past, you might need additional vaccines before starting ULTOMIRIS. Your healthcare provider will decide if you need additional meningococcal vaccines.
  5. 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 meningococcal infection: fever, fever with high heart rate, headache and fever, confusion, muscle aches with flu-like symptoms, fever and a rash, headache with nausea or vomiting, headache with a stiff neck or stiff back, or eyes sensitive to light.

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 ULTOMIRIS dose. Your risk of meningococcal infection may continue for several months after your last dose of ULTOMIRIS. It is important to show this card to any healthcare provider who treats you. This will help them diagnose and treat you quickly.

ULTOMIRIS is only available through a program called the ULTOMIRIS and SOLIRIS Risk Evaluation and Mitigation Strategy (REMS). Before you can receive ULTOMIRIS, 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 are not up to date on your vaccines; give you a Patient Safety Card about your risk of meningococcal infection.

ULTOMIRIS may also increase the risk of other types of serious infections, including Streptococcus pneumoniae, Haemophilus influenzae, and Neisseria gonorrhoeae. Your child should receive vaccines against Streptococcus pneumoniae and Haemophilus influenzae type b (Hib) if treated with ULTOMIRIS. Certain people may be at risk of serious infections with gonorrhea.

Who should not receive ULTOMIRIS?

Do not receive ULTOMIRIS if you have a serious meningococcal infection when you are starting ULTOMIRIS.

Before you receive ULTOMIRIS, tell your healthcare provider about all of your medical conditions, including if you: have an infection or fever, are pregnant or plan to become pregnant, and are breastfeeding or plan to breastfeed. It is not known if ULTOMIRIS will harm your unborn baby or if it passes into your breast milk. You should not breastfeed during treatment and for 8 months after your final dose of ULTOMIRIS.

Tell your healthcare provider about all the vaccines you receive and medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements which could affect your treatment.

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 ULTOMIRIS. Stopping ULTOMIRIS may cause 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, tiredness, blood in your urine, stomach-area (abdomen) pain, shortness of breath, blood clots, trouble swallowing, and erectile dysfunction (ED) in males.

What are the possible side effects of ULTOMIRIS?

ULTOMIRIS can cause serious side effects including infusion-related reactions. Symptoms of an infusion-related reaction with ULTOMIRIS 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 treatment of ULTOMIRIS and tell your healthcare provider right away if you develop these symptoms, or any other symptoms during your ULTOMIRIS infusion that may mean you are having a serious infusion-related reaction, including: chest pain, trouble breathing or shortness of breath, swelling of your face, tongue, or throat, and feel faint or pass out.

The most common side effects of ULTOMIRIS in people treated for PNH are upper respiratory tract infection and headache.

Tell your healthcare provider about any side effect that bothers you or that does not go away. These are not all the possible side effects of ULTOMIRIS. For more information, ask your healthcare provider or pharmacist. Call your healthcare provider right away if you miss an ULTOMIRIS infusion or for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

INDICATION

What is ULTOMIRIS?

ULTOMIRIS is a prescription medicine used to treat adults and children 1 month of age and older with a disease called Paroxysmal Nocturnal Hemoglobinuria (PNH).

It is not known if ULTOMIRIS is safe and effective in children younger than 1 month of age.

Please see the full Prescribing Information and Medication Guide for ULTOMIRIS, including Boxed WARNING regarding serious meningococcal infections.