Experience immediate and complete control for up to 8 weeks.a
Many clinical trials have established the short- and long-termb efficacy for starting or switching to ULTOMIRIS in people diagnosed with PNH.
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).
bThe primary evaluation period of the clinical trials was 26 weeks, and the extension period of the trials had a range of 3 to 5 years.
ULTOMIRIS was studied in the largest
PNH clinical trial program to date
ULTOMIRIS is the first and only FDA-approved medication
for both
children and adults with PNHc
cOne 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
Adults who received ULTOMIRIS and had no prior PNH treatment experienced a reduction of PNH signs and symptoms
74%
(92 out of 125 adults)
did not require blood transfusions, which reflects how well hemolysis is being controlled
54%
(67 out of 125 adults)
had normalized LDH levels
On average, there was a 76.8% reduction in LDH levels from the study start
Low or normalized LDH levels are associated with control over intravascular hemolysis
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 blockingC5 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
Breakthrough hemolysis is red blood cell destruction that happens despite ongoing treatment
98.4%
(123 out of 125 adults)
were free of blood clots, which indicates how well PNH-induced clotting is being controlled
68%
(85 out of 125 adults)
had stable hemoglobin levels, which can mean that anemia is under control
Decreased fatigue
The average FACIT-Fatigue score improved by an average of 7.1 points during the study. Scores on the scale range from 0 to 52, with higher scores indicating less fatigue
The most common side effects (≥10%) of ULTOMIRIS in adults with PNH were upper respiratory tract infection and headache. These primary and secondary outcomes in adults stay consistent through 1 year of treatment with ULTOMIRIS. A corresponding trial of SOLIRIS-experienced patients showed similar results.
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.
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).
FACIT=Functional Assessment of Chronic Illness Therapy; LDH=lactate dehydrogenase; C5=a protein within the complement system.
For patients in the trial previously on SOLIRIS, the results showed:
Low LDH levels
were maintained
0.8% average reduction in LDH levels
Low or normalized LDH levels are associated with control over intravascular hemolysis
88%
(85 out of 97 adults)
did not need blood transfusions, which reflects how well hemolysis is being controlled
100%
(97 out of 97 adults)
had lower levels of free C5 indicating immediate, complete, and long-term C5 inhibition
This measure shows how well ULTOMIRIS is blocking C5 activity
100%
(97 out of 97 adults)
were free of breakthrough hemolysis events
Breakthrough hemolysis is red blood cell destruction that happens despite ongoing treatment
100%
(97 out of 97 adults)
were free of blood clots which indicates how well PNH-induced clotting is being controlled
76%
(74 out of 97 adults)
had stable hemoglobin levels, which can mean that anemia is under control
Fatigue levels stayed low
in these patients whose PNH was already controlled on SOLIRIS. The average FACIT-Fatigue score improved by an average of approximately 2.6 points over the course of the study
The most common side effects (≥10%) of ULTOMIRIS in adults with PNH were upper respiratory tract infection and headache. These primary and secondary outcomes in adults stayed consistent through 1 year of treatment with ULTOMIRIS.
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.
FACIT=Functional Assessment of Chronic Illness Therapy; LDH=lactate dehydrogenase; C5=a protein within the complement system.
Children who received ULTOMIRIS experienced a reduced risk of PNH signs and symptoms
100%
(13 out of 13 children)
had levels of free C5 indicating immediate, complete, and long-term C5 inhibition
This measure shows how well ULTOMIRIS is blocking C5 activity
85%
(11 out of 13 children)
did not need blood transfusions, which reflects how well hemolysis is being controlled
69%
(9 out of 13 children)
had stable hemoglobin levels, which can mean that anemia is under control
100%
(13 out of 13 children)
were free of breakthrough hemolysis events
Breakthrough hemolysis is red blood cell destruction that happens despite ongoing treatment
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 not 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 (>20%) 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; C5=a protein within the complement system.
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.
- You must complete or update meningococcal vaccine(s) at least 2 weeks before your first dose of ULTOMIRIS.
- 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.
- 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.
- 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.
- 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. It is not known if ULTOMIRIS will harm your unborn baby.
- Pregnancy Registry: There is a registry for pregnant women who take ULTOMIRIS to check the health of the pregnant mother and her baby. If you are pregnant or become pregnant while taking ULTOMIRIS, talk to your healthcare provider about how you can join this registry or you may contact the registry at 1-833-793-0563 or www.UltomirisPregnancyStudy.com to enroll.
- 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 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, stomach (abdominal) pain, muscle spasms, changes in blood pressure, tiredness, feeling faint, shaking chills (rigors), discomfort in your arms or legs, or bad taste. 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.