ULTOMIRIS® (ravuizumab-cwvz) injection for intravenous use 300 mg/30 mL vial
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FIRST, UNDERSTAND PNH,
THEN LEARN TO MANAGE IT

Living with a rare disease like paroxysmal nocturnal hemoglobinuria (PNH) can be a challenge, but the more you know about PNH, the better you may be able to manage it.

What does PNH mean?

P
paroxysmal
sudden attack, from time to time
N
nocturnal
at nighttime
H
hemoglobinuria
hemoglobin in the urine

In PNH, a change occurs in the bone marrow stem cells, altering how red blood cells (RBCs) are made. The stem-cell change causes fewer normal cells to be made and the potentially lifelong production of “bad” cells, or PNH cells. These PNH cells are missing important protective proteins. Without these proteins, one of your body’s natural defense systems, called “complement,” destroys PNH RBCs. This destruction is known as hemolysis, the main cause of major health problems in PNH, including some that may be life-threatening. If you have PNH, you are at constant risk of hemolysis.

In some patients, in addition to making PNH cells, bone marrow may also have trouble simply making cells.

1
PNH RBC missing
protective proteins

Hemolysis

  • Hemo—blood
  • Lysis—breaking apart of cells
2
Complement
attack
3
RBC destruction—
hemolysis

Hemolysis in PNH

  • Is a process of red blood cell destruction by complement system
  • Is an ongoing process
  • Sometimes happens at high rates
  • Is the cause of signs, symptoms, and potentially serious health problems

Did you know?

PNH is rare For every one million people, there are only 1.3 new cases of PNH each year
PNH can affect Men & Women of all ages, regardless of race
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What do the lab results mean?

Your doctor may order some of the following lab tests to find out if you have PNH and to monitor your condition:

Evidence of elevated hemolysis with this test
Lactate dehydrogenase (LDH) level
Measures LDH, 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.
Signs of kidney damage with this test
Creatinine
Measures creatinine, a waste product in the blood, to show how well your kidneys are working. The creatinine level can help reflect if and how PNH is affecting your kidneys.
Platelet levels with this test
Platelet count
Measures the number of platelets in your blood. Platelets are used for clotting and play an important role in helping you heal from injury. PNH may also affect your platelet level.
Clone size with this test
High-sensitivity flow cytometry
Measures the actual number of red and white blood cells affected by PNH in a small sample of circulating blood taken from your arm. This is the standard test for confirming whether or not you have PNH. Through continued monitoring, your doctor can tell if your clone size is changing.
The tests listed in the table above are just some that your doctor might order. There may be others. Work closely with your doctor and keep track of your results, too.

What is clone size?

Clone size, which can be measured by high-sensitivity flow cytometry, is the percentage of blood cells in your body that have been affected by PNH and, therefore, do not have the protective proteins that blood cells usually have on the surface.

Many of your blood cells may be normal, but anyone with PNH will have some clones. A larger clone size means you have more blood cells that are missing protective proteins. But even small clone sizes can lead to PNH-related health problems—a small clone size does not necessarily mean that you have less PNH. Your clone size may change over time, and symptoms can get worse over time if PNH is left unmanaged. That is why continued monitoring and management are very important.

The difference between RBC and white blood cell (WBC) clone sizes can indicate the extent of hemolysis
RBC CLONE SIZE
PNH CELLS
WBC CLONE SIZE
(TRUE CLONE SIZE)
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PNH can be tough to identify, as its symptoms are similar to those of other diseases.

You may not see what’s happening below the surface, and even when you feel fine, lab results can reveal potential problems.

The signs and symptoms of PNH may not always be obvious, so it’s important to discuss what you notice with your doctor.

Symptoms you may see

Signs you may not see

COMMON SIGNS AND SYMPTOMS OF PNH

Symptoms you may
see or feel:

  • Fatigue
  • Pain
  • Dark-colored urine
  • Shortness of breath
  • Difficulty swallowing
  • Yellowing of the skin and eyes
  • Erectile dysfunction (ED)

Signs you may not
see or feel:

  • Blood clots
  • Kidney disease
  • Damage to your organs
  • Stroke
  • Heart attack
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Your doctor will consider all test results, signs, and symptoms

How can I help my doctor monitor my PNH?

Track your signs, symptoms, and lab results. They will show you and your doctor 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 physician 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 doctor 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 doctor about treatment options—you shouldn’t have to feel like feeling sick is normal.

Speaking with your doctor is key to successful management of PNH

Asking questions will keep you informed. Here are a few you might want to ask your doctor:

  • Can my disease get worse over time?
  • How will I know if my PNH is getting worse or better?
  • I would like a copy of my lab results. Would you please help me understand them?
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Talk to your doctor about ULTOMIRIS

Talk to your doctor about long-acting ULTOMIRIS for extended control of your PNH between infusions.

MAKING THE SWITCH

PNH may be rare, but you’re not alone

OneSource™ can help you learn about PNH, co-verify your insurance coverage, and identify helpful resources for people living with PNH and those who care for them.

LEARN MORE ABOUT ONESOURCE

INDICATION & IMPORTANT SAFETY INFORMATION for ULTOMIRIS® (ravulizumab‑cwvz), INCLUDING BOXED WARNING

INDICATION

What is ULTOMIRIS?
ULTOMIRIS is a prescription medicine called a monoclonal antibody. ULTOMIRIS is used to treat:

  • adults with a disease called Paroxysmal Nocturnal Hemoglobinuria (PNH).

It is not known if ULTOMIRIS is safe and effective in children with PNH.

IMPORTANT SAFETY INFORMATION

What is the most important information I should know about ULTOMIRIS?
ULTOMIRIS is a medicine that affects your immune system. ULTOMIRIS can lower the ability of your immune system to fight infections.

  • ULTOMIRIS increases your chance of getting serious and life-threatening meningococcal infections. Meningococcal infections may quickly become life-threatening and cause death if not recognized and treated early.
    1. You must receive meningococcal vaccines at least 2 weeks before your first dose of ULTOMIRIS if you have not already had this vaccine.
    2. If your doctor decided that urgent treatment with ULTOMIRIS is needed, you should receive meningococcal vaccination as soon as possible.
    3. If you have not been vaccinated and ULTOMIRIS therapy must be initiated immediately, you should also receive 2 weeks of antibiotics with your vaccinations.
    4. If you had a meningococcal vaccine in the past, you might need additional vaccination before starting ULTOMIRIS. Your doctor will decide if you need additional meningococcal vaccination.
    5. Meningococcal vaccines reduce the risk of meningococcal infection but do not prevent all meningococcal infections. Call your doctor or get emergency medical care right away if you get any of these signs and symptoms of a meningococcal infection:
      • headache with nausea or vomiting
      • headache and fever
      • headache with a stiff neck or stiff back
      • fever
      • fever and a rash
      • confusion
      • muscle aches with flu-like symptoms
      • eyes sensitive to light

Your doctor will give you a Patient Safety Card about the risk of 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 doctor or nurse who treats you. This will help them diagnose and treat you quickly.

ULTOMIRIS is only available through a program called the ULTOMIRIS REMS. Before you can receive ULTOMIRIS, your doctor must:

  • enroll in the ULTOMIRIS REMS program
  • counsel you about the risk of meningococcal infection
  • give you information about the symptoms of meningococcal infection
  • give you a Patient Safety Card about your risk of meningococcal infection, as discussed above
  • make sure that you are vaccinated with a meningococcal vaccine

ULTOMIRIS may also increase the risk of other types of serious infections.

  • People who take ULTOMIRIS may have an increased risk of getting infections caused by Streptococcus pneumoniae and Haemophilus influenzae.
  • Certain people may also have an increased risk of gonorrhea infection. Talk to your doctor to find out if you are at risk for gonorrhea infection, about gonorrhea prevention, and regular testing.

Call your doctor right away if you have any new signs or symptoms of infection.

Who should not receive ULTOMIRIS?
Do not receive ULTOMIRIS if you:

  • have a meningococcal infection
  • Have not been vaccinated against meningococcal infection unless your doctor decides that urgent treatment with ULTOMIRIS is needed. See “What is the most important information I should know about ULTOMIRIS.”

Before you receive ULTOMIRIS, tell your doctor about all of your medical conditions, including if you:

  • have an infection or fever.
  • are pregnant or plan to become pregnant. It is not known if ULTOMIRIS will harm your unborn baby.
  • are breastfeeding or plan to breastfeed. It is not known if ULTOMIRIS passes into your breast milk. You should not breastfeed during treatment and for 8 months after your final dose of ULTOMIRIS.

Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. ULTOMIRIS and other medicines can affect each other causing side effects.

Know the medicines you take and the vaccines you receive. Keep a list of them to show your doctor and pharmacist when you get a new medicine.

How should I receive ULTOMIRIS?

  • ULTOMIRIS is given through a vein by intravenous (I.V.) infusion usually over about 2 hours in adults.
  • If you are an adult with PNH, you will usually receive:
    • a starting dose of ULTOMIRIS as an infusion by your doctor, and then
    • 2 weeks later, you will start to receive an infusion of ULTOMIRIS every 8 weeks.
  • If you are changing treatment from SOLIRIS to ULTOMIRIS, you should receive your starting dose of ULTOMIRIS 2 weeks after your last dose of SOLIRIS.
  • After each infusion, you should be monitored for at least 1 hour for infusion reactions. See “What are the possible side effects of ULTOMIRIS?”
  • If you have PNH and you stop receiving ULTOMIRIS, your doctor 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
    • erectile dysfunction (ED) in males
  • If you miss an ULTOMIRIS infusion, call your doctor right away.

What are the possible side effects of ULTOMIRIS?
ULTOMIRIS can cause serious side effects including:

  • See “What is the most important information I should know about ULTOMIRIS?”
  • Infusion reactions. Infusion reactions may happen during your ULTOMIRIS infusion. Symptoms of an infusion reaction with ULTOMIRIS may include lower back pain, pain with the infusion, feeling faint or discomfort in your arms or legs. Tell your doctor or nurse right away if you develop these symptoms, or any other symptoms during your ULTOMIRIS infusion that may mean you are having a serious infusion reaction, including:
    • chest pain
    • trouble breathing or shortness of breath
    • swelling of your face, tongue, or throat
    • feel faint or pass out
    Your doctor will treat your symptoms as needed.

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

Tell your doctor 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 doctor or pharmacist.

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Please see the accompanying full Prescribing Information and Medication Guide for ULTOMIRIS, including Boxed WARNING regarding serious and life-threatening meningococcal infections/sepsis.