ULTOMIRIS, the #1 treatment* for adults with PNH, is NOW FDA APPROVED for children. Learn More.
*Based on US market share data.

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ULTOMIRIS is designed for long-acting control of C5, intravascular hemolysis, and PNH symptoms

Developed out of Alexion’s commitment to patients with PNH, ULTOMIRIS is the first and only long-acting medication approved by the FDA to treat PNH.

ULTOMIRIS just once every 4 or 8 weeksa

ULTOMIRIS needs to be infused as few as 6-7 times a yearb—that’s up to 20 fewer infusions than with SOLIRIS® (eculizumab).

aStarting 2 weeks after the initial loading dose, maintenance doses are administered every 8 weeks for adults and every 4 or 8 weeks for pediatric patients (depending on body weight).

bFollowing loading dose.

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.c 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.

cStarting 2 weeks after the initial loading dose, maintenance doses are administered every 8 weeks for adults and every 4 or 8 weeks for pediatric patients (depending on body weight).


Why ULTOMIRIS?

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 patients taking SOLIRIS
  • 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 patients 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 patients in the ULTOMIRIS group experienced breakthrough hemolysis vs 5.1% in the SOLIRIS group.

ULTOMIRIS was shown to be effective for up to
8 weeks following maintenance dosing in clinical trials

ULTOMIRIS starts working at the
time of your first infusion and
keeps working until it’s time for
your next infusion.
ULTOMIRIS is dosed
based on your weight.

Do not start ULTOMIRIS if you have a meningococcal infection.

ULTOMIRIS was studied in the largest-ever
PNH clinical trial program

ULTOMIRIS is the first and only FDA-approved medication
for both children and adults with PNHd

dOne month of age and older.

The ULTOMIRIS clinical studies each lasted
6 months and included…

246 adults who had no prior
PNH treatment
with a complement inhibitor
called SOLIRIS® (eculizumab)

195 adults who had received
treatment with SOLIRIS

13 children 8 who had received
prior PNH
treatment
with SOLIRIS and 5 who had not


The effects of ULTOMIRIS on PNH were measured in several different ways, including…

Amount of free C5
in the blood

Low levels mean that C5 is
being blocked by ULTOMIRIS

Amount of LDH
in the blood

Low or normalized LDH
levels are associated with
control over intravascular hemolysis

Rate of transfusion
avoidance

The number of people who
did not need transfusions
during the study can
reflect how well hemolysis
is being controlled

Rate of breakthrough
hemolysis

Breakthrough hemolysis is red
blood cell destruction that happens
despite ongoing treatment

Breakthrough hemolysis events were defined as experiencing at least 1 new or worsening
sign or symptom of hemolysis that occurs along with elevated LDH levels
(after LDH levels were previously reduced through treatment)

Rate of adverse
events involving
blood vessels

The number of adults who
had adverse events involving
blood vessels—which included
blood clots—in the study
can indicate how well
PNH-induced clotting is
being controlled

Hemoglobin
stabilization

Stable levels of
hemoglobin in the blood
can mean that anemia
is under control

Fatigue

Fatigue was measured using
a scale called FACIT-Fatigue.
Scores on the scale range
from 0 to 52, with higher
scores indicating less fatigue

The reported fatigue in these
studies may be an underestimation
or overestimation

FACIT=Functional Assessment of Chronic Illness Therapy; LDH=lactate dehydrogenase.


ULTOMIRIS reduced the risk of PNH
signs and symptoms

In adults who received ULTOMIRIS and had
no prior PNH treatment…

74%

(92 out of 125 adults)

did not need blood
transfusions

54%

(67 out of 125 adults)

had normalized LDH levels

On average, there was a 76.8% reduction in LDH levels from the starting point of the study

100%

(125 out of 125 adults)

had levels of free C5 that
indicated immediate, complete,
and sustained C5 inhibition

This measure shows how well ULTOMIRIS is working to block C5 activity

96%

(120 out of 125 adults)

were free of breakthrough
hemolysis events

The 5 breakthrough events that happened in the study were associated with an
infection or an unknown cause, not elevated free C5 levels

98.4%

(123 out of 125 adults)

were free of
blood clots

68%

(85 out of 125 adults)

had stable
hemoglobin levels

Decreased fatigue

The average FACIT-Fatigue
score improved by an
average of 7.1 points
over
the course of the study

The average score for the adult
general population has been estimated to be 43.5

There was no observable difference in fatigue between ULTOMIRIS and SOLIRIS after 26 weeks of treatment compared to baseline as measured by the FACIT-Fatigue instrument. Patient-reported fatigue may be an underestimation or overestimation, because patients were not blinded to treatment assignment

The most frequent adverse reactions (≥10%) with ULTOMIRIS were respiratory tract infection and headache.

These effects in adults stayed consistent through 1 year of treatment with ULTOMIRIS. A corresponding trial of SOLIRIS-experienced patients showed similar results.


ULTOMIRIS reduced the risk of PNH
signs and symptoms

In adults who received ULTOMIRIS and whose PNH was
well controlled on SOLIRIS® (eculizumab)…

Low LDH levels
were maintained

There was a 0.8% average
reduction in LDH levels

88%

(85 out of 97 adults)

did not need blood
transfusions

100%

(97 out of 97 adults)

had levels of free C5 that indicated
immediate, complete, and
sustained C5 inhibition

This measure shows how well ULTOMIRIS is working to block C5 activity

100%

(97 out of 97 adults)

were free of breakthrough
hemolysis events

100%

(97 out of 97 adults)

were free of
blood clots

76%

(74 out of 97 adults)

had stable
hemoglobin levels

Fatigue levels
stayed low

in these patients whose PNH was already controlled on SOLIRIS. The average FACIT-Fatigue score improved by an average of 2.0 points over the course of the study

The average score for the adult general population has been estimated to be 43.5

There was no observable difference in fatigue between ULTOMIRIS and SOLIRIS after 26 weeks of treatment compared to baseline as measured by the FACIT-Fatigue instrument. Patient-reported fatigue may be an underestimation or overestimation, because patients were not blinded to treatment assignment

These effects in adults stayed consistent through 1 year of treatment with ULTOMIRIS.


ULTOMIRIS reduced the risk of PNH signs and symptoms

In children who received ULTOMIRIS…

100%

(13 out of 13 children)

had levels of free C5 that indicated immediate, complete, and sustained C5 inihibition

This measure shows how well ULTOMIRIS is working to block C5 activity

85%

(11 out of 13 children)

did not need blood
transfusions

69%

(9 out of 13 children)

had stable
hemoglobin levels

100%

(13 out of 13 children)

were free of breakthrough
hemolysis events

48%

Average reduction
in LDH levels

In the 5 children who had
not received prior PNH
treatment with SOLIRIS

4.7%

Average increase
in LDH levels

In the 8 children who
had received prior PNH
treatment with SOLIRIS

Decreased fatigue

All 5 children who had received prior PNH treatment with SOLIRIS had a clinically relevant decrease in fatigue over the 26-week study, as measured by Pediatric FACIT-Fatigue

The 8 children who had received prior PNH treatment with SOLIRIS also had a slight decrease in fatigue

The most common side effects of ULTOMIRIS in children with PNH were upper respiratory tract infection, anemia, abdominal pain, and headache.

FACIT=Functional Assessment of Chronic Illness Therapy; LDH=lactate dehydrogenase.


Switching to ULTOMIRIS from SOLIRIS (eculizumab)

When you switch to ULTOMIRIS from SOLIRIS:

  • 2 weeks after your final eculizumab dose, your doctor will administer what’s known as a “loading dose”
    • This will only occur when you begin treatment with ULTOMIRIS
  • 2 weeks after you receive the loading dose, you will receive your first “maintenance dose,” the dose at which you will continue to receive ULTOMIRIS up to once every 8 weekse

eStarting 2 weeks after the initial loading dose, maintenance doses are administered every 8 weeks for adults and every 4 or 8 weeks for pediatric patients (depending on body weight).

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What do I need to know before taking ULTOMIRIS?

Infusion-related reactions

Infusion-related reactions may happen during your ULTOMIRIS infusion. Symptoms of an infusion-related reaction with ULTOMIRIS may include lower back pain, 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.

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
ULTOMIRIS is the only long-acting PNH medication approved by the FDA.

Less infusions means more freedom

ULTOMIRIS, administered up to every 8 weeks,f gives people with PNH the possibility of more freedom and flexibility between treatments.

ULTOMIRIS is given through a vein by intravenous (IV) infusion.

If you are an adult with PNH
you will usually receive an infusion of ULTOMIRIS every 8 weeks that begins 2 weeks after your starting dose

If you are a child with PNH
you will usually receive an infusion of ULTOMIRIS every 4 or 8 weeks, depending on your body weight, that begins 2 weeks after your starting dose

After each infusion, you should be monitored for at least 1 hour for infusion-related reactions.


fStarting 2 weeks after the initial loading dose, maintenance doses are administered every 8 weeks for adults and every 4 or 8 weeks for pediatric patients (depending on body weight).


Infusion tips

You might be feeling unsure about getting intravenous infusions, but there are ways to improve the experience:

  • Drink plenty of water. This will help your doctor find your veins more easily

  • Wear comfortable, layered clothing that you can adjust in case you become overly warm or cool

  • Keep busy during your infusion by reading, watching TV, or doing any other activity you can do while seated and remaining still

You may need to arrive early or stay late after your treatment, depending on the requirements of your treatment center.

Long-acting control of intravascular hemolysis (IVH) means more time between infusions.

You must receive a meningococcal vaccine at least 2 weeks before your first dose of ULTOMIRIS if you have not already had this vaccine

Get vaccinated.

ULTOMIRIS can lower the ability of your immune system to fight some infections. Before taking ULTOMIRIS, you must be vaccinated against meningococcal infection, a severe infection that can occur in the blood and that requires immediate medical attention. Your doctor or nurse will make sure you receive this vaccine at least 2 weeks before your first infusion.

If your doctor decided that urgent treatment with ULTOMIRIS is needed, you should get the meningococcal vaccine as soon as possible.

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.

What are the symptoms of meningococcal infection?

The same mechanism that ULTOMIRIS uses to control hemolysis can increase your risk of getting an infection, especially a meningococcal infection. Call your doctor or get emergency medical care right away if you get any of these signs or 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

Carry your Patient Safety Information Card NOW.

The Patient Safety Information Card lists the signs and symptoms of a meningococcal infection and tells you what to do if you experience any of them.

Start carrying the card today, and 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 weeks 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.


How it works in adults: ULTOMIRIS in the body

How it works in adults: ULTOMIRIS in the body

While ULTOMIRIS and SOLIRIS both inhibit the complement protein C5, ULTOMIRIS has been engineered to have a ~4x longer half-life (the time it takes your body to remove half of the drug).

Lab tests that your doctor 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 doctor 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 doctor 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

Sticking to your prescribed treatment schedule

For ULTOMIRIS to effectively reduce hemolysis, the drug needs to stay above a certain level in your blood. However, like all drugs, ULTOMIRIS is broken down and removed from your body over time.

The time that it takes your body to remove half of the drug is called the “half-life” of that drug. A regular therapy schedule keeps ULTOMIRIS in your body at a level where it works best.

ULTOMIRIS should be infused according to the recommended dosing schedule for you to get the most out of your treatment. If the level of ULTOMIRIS in your body gets too low, hemolysis can occur.

Hemolysis is the underlying cause of major health problems in PNH. Missing doses can allow hemolysis to happen.

Work closely with your healthcare team to keep track of your infusions, and check in with your doctor regularly to best manage your PNH.

If you miss an ULTOMIRIS infusion, call your doctor right away.
To get the most from your ULTOMIRIS therapy, stick with your treatment schedule.
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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 can lower the ability of your immune system to fight infections.

  • ULTOMIRIS increases your chance of getting serious and life-threatening meningococcal infections that 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 are not vaccinated.
  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. Your doctor will decide if you need additional vaccination.
  5. Meningococcal vaccines reduce 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 and 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. It is important to show this card to any doctor or nurse to 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 and a Patient Safety Card about the symptoms and your risk of meningococcal infection (as discussed above); and make sure that you are vaccinated with a meningococcal vaccine, and if needed, get revaccinated with the meningococcal vaccine. Ask your doctor if you are not sure if you need to be revaccinated.

ULTOMIRIS may also increase the risk of other types of serious infections. Make sure your child receives vaccinations against Streptococcus pneumoniae and Haemophilus influenzae type b (Hib) if treated with ULTOMIRIS. 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 or have not been vaccinated against meningococcal infection unless your doctor decides that urgent treatment with ULTOMIRIS is needed.

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