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NOW APPROVED for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD).1

About ULTOMIRIS

Select an indication to view relevant mechanism of action information.

gMG Atypical-HUS
& PNH

Indications:

ULTOMIRIS is indicated for the treatment of adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive.

ULTOMIRIS is indicated for the treatment of adult and pediatric patients one month of age and older with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA).

Limitation of Use: ULTOMIRIS is not indicated for the treatment of patients with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS).

ULTOMIRIS is indicated for the treatment of adult and pediatric patients one month of age and older with paroxysmal nocturnal hemoglobinuria (PNH).

Resources and support

Select an indication to view relevant treatment resources and support documents.

gMG Atypical-HUS
& PNH

Indications:

ULTOMIRIS is indicated for the treatment of adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive.

ULTOMIRIS is indicated for the treatment of adult and pediatric patients one month of age and older with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA).

Limitation of Use: ULTOMIRIS is not indicated for the treatment of patients with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS).

ULTOMIRIS is indicated for the treatment of adult and pediatric patients one month of age and older with paroxysmal nocturnal hemoglobinuria (PNH).

ULTOMIRIS pediatric efficacy and safety

ULTOMIRIS, the first and only long-acting complement inhibitor for atypical-HUS, provided immediate, complete, and sustained complement inhibition in adult and pediatric patients 1 month of age and older.1

For treatment of atypical-HUS in adult and pediatric patients 1 month of age and older. Not indicated in STEC‑HUS.1

STEC-HUS=Shiga toxin–producing E coli hemolytic uremic syndrome.

Actor portrayal

Immediate complement inhibition

ULTOMIRIS provided immediate, complete, and sustained complement inhibition1-3

A total of 14 pediatric patients with atypical-HUS who were eculizumab naïve were evaluated for efficacy. The study consisted of a 26-week initial evaluation period, followed by an extension period up to 4.5 years, with an interim analysis at 50 weeks.

See full study design

Immediate

100% demonstrated
C5 inhibition

14 of 14 pediatric patients in a 26-week study demonstrated complete C5 inhibition after the first infusion of ULTOMIRISa

Complete

71% achieved complete
TMA response

10 of 14 pediatric patients (95% CI: 42%-92%) met the composite endpoint of complete TMA responseb with ULTOMIRIS by 26 weeks, which was maintained through 50 weeks

Sustained

C5 inhibition was sustained
for 4 or 8 weeks, with
the possibility to live
dialysis-free

aAs measured by free C5 serum concentration of <0.5 mcg/mL.1

bComplete TMA response was defined as normalization of hematological parameters (platelet count and LDH) and ≥25% improvement in serum creatinine from baseline during the 26-week initial evaluation period. Patients had to meet all criteria at 2 separate assessments obtained at least 4 weeks (28 days) apart and any measurement in between.1

C5=complement protein 5; LDH=lactate dehydrogenase; TMA=thrombotic microangiopathy.

Complete TMA response

Nearly 3 out of 4 pediatric patients (n=10/14) taking ULTOMIRIS achieved complete TMA response, which was maintained by all patients at Week 501,3,c

See full study design



Median time to complete TMA response was 30.0 days (95% CI: 22.0, 88.0).3


Complete TMA response

Children

26 weeks
71%
(95% Cl: 42%-92%) n=10/14
50 weeks
94%
n=17/18

Median time to complete TMA response was 30.0 days (95% CI: 22.0, 88.0).3

The interim analysis at 26 weeks was performed with 14 pediatric patients with atypical-HUS. As the study was ongoing, additional patients were recruited, and the 50-week analysis included 18 pediatric patients.

cComplete TMA response was defined as normalization of hematological parameters (platelet count and LDH) and ≥25% improvement in serum creatinine from baseline during the 26-week initial evaluation period. Patients had to meet all criteria at 2 separate assessments obtained at least 4 weeks (28 days) apart and any measurement in between.1 Patients that did not have a response were censored at the date of last visit or study discontinuation at the time when the analysis was performed.2

BL=baseline; LDH=lactate dehydrogenase; TMA=thrombotic microangiopathy.



Components of complete TMA response1,3,d


Platelet count normalization

Children

week 26
93%
(95% Cl: 66%-99%) n=13/14
week 50
94%
n=17/18

LDH normalization

Children

week 26
86%
(95% Cl: 57%-98%) n=12/14
week 50
94%
n=17/18

≥25% Improvement in serum creatinine from baseline

Children

week 26
79%
(95% Cl: 49%-95%) n=11/14
week 50
94%
n=17/18

Hematologic normalization

Children

week 26
86%
(95% Cl: 57%-98%) n=12/14
week 50
94%
n=17/18

dComplete TMA response during the 26-week initial evaluation period was summarized by number and proportion of complete TMA responders (with 2-sided 95% confidence interval). Statistical analysis was not performed on 50-week data.

LDH=lactate dehydrogenase; TMA=thrombotic microangiopathy.


99.6% of all free C5 serum samples in pediatric patients showed complete inhibition of C5 throughout the 6-month study period with ULTOMIRIS2,e

eAs measured by free C5 serum concentration of <0.5 mcg/mL.1

C5=complement protein 5.

Changes in CKD stage

ULTOMIRIS offers 4 or 8 weeks of sustained C5 inhibition and an opportunity to discontinue dialysis in pediatric patients1,3

Patients with chronic kidney disease (CKD) Stage 5 cannot worsen and may not be able to improve2

See full study design

CKD stage shift from baseline to end of initial evaluation period (full analysis set)2,f
Post-baseline CKD stage at Day 183 (N=13)g
Baseline CKD
stage
1
n (%)
2
n (%)
3A
n (%)
3B
n (%)
4
n (%)
5
n (%)
1 (n=0)
0
0
0
0
0
0
2 (n=2)
1 (7.7)
0
0
0
0
0
3A (n=1)
1 (7.7)
0
0
0
0
0
3B (n=0)
0
0
0
0
0
0
4 (n=6)
3 (23.1)
1 (7.7)
1 (7.7)
0
1 (7.7)
0
5 (n=5)
3 (23.1)
1 (7.7)
0
0
0
1 (7.7)
Total (n=14)
8 (61.5)
2 (15.4)
1 (7.7)
0
1 (7.7)
1 (7.7)

fGreen shading indicates improvement vs baseline; orange shading indicates worsening vs baseline. CKD stage classified based on the National Kidney Foundation CKD Stage (eGFR values were: Stage 1: ≥90; Stage 2: 60 to 89; Stage 3A: 45 to 59; Stage 3B: 30 to 44; Stage 4: 15 to 29; Stage 5: <15 [including dialysis]). The baseline was derived based on last available eGFR before starting treatment. Patients with both baseline and at least 1 value at post-baseline visits were included in the summary. Percentages based on number of patients with non-missing data at both baseline and post-baseline visit.

gThe percentages for the post-baseline CKD stage at Day 183 are based on 13 patients with available data.

C5=complement protein 5; CKD=chronic kidney disease; eGFR=estimated glomerular filtration rate.

Discontinuing dialysis may be possible

80% of pediatric patients (n=4/5) who required dialysis at study entry discontinued dialysis after the first month in the study.1

No patient started dialysis during the study.1

100% of pediatric patients (n=6/6) who required dialysis at study entry discontinued dialysis
by Week 50.3

Study design

ULTOMIRIS was evaluated in a 26-week study in pediatric patients with atypical-HUS, followed by an ongoing extension study1,3

Pivotal study and extension study1,3



hULTOMIRIS weight-based dosing regimen.

Select inclusion criteria:

  • Platelet count ≤150 x 109 cells/L
  • Evidence of hemolysis (eg, elevated serum LDH, serum creatinine ≥97.5%, or required dialysis)

Select exclusion criteria:

  • Patients with TMA due to ADAMTS13 deficiency, STEC‑HUS, and genetic defect in cobalamin C metabolism

Pivotal study

  • The pivotal study was conducted in 16 pediatric patients. A total of 14 eculizumab-naïve patients with documented diagnosis of atypical-HUS were enrolled and included in the interim 26-week analysis.

Extension study

  • Pediatric patients with atypical-HUS in the 26-week initial evaluation period could enter an ongoing 4.5-year extension period.
  • Long-term extension data are from an interim analysis with 18 patients at Week 50.

ADAMTS13=a disintegrin and metalloproteinase with a thrombospondin type 1 motif member 13; LDH=lactate dehydrogenase; STEC-HUS=Shiga toxin–producing E coli hemolytic uremic syndrome; TMA=thrombotic microangiopathy.

Select baseline characteristics1,2

Select demographics and baseline characteristics (N=14)
Age at time of first infusion (years) category, n (%)
Birth to <2 years
2 (14.3)
2 to <6 years
7 (50.0)
6 to <12 years
4 (28.6)
12 to <18 years
1 (7.1)
Sex
Female, n (%)
9 (64.3)
Race, n (%)i
White
7 (50.0)
Asian
4 (28.6)
Black or African American
2 (14.3)
American Indian or Alaskan Native
1 (7.1)
Unknown or other
1 (7.1)
Stage 5 CKD assessed by eGFR, n (%)
5 (35.7)
History of prior kidney transplant, n (%)
1 (7)
Extrarenal signs or symptoms of atypical-HUS at baseline, n (%)
10 (71)
Mean platelet count [normal range 229 to 533 x 109/L]
60.50 x 109/L
Mean LDH in serum [normal range 165 to 395 U/L]
2324.11 U/L
Mean eGFR [normal range ≥60 mL/min/1.73 m2]
28.4 mL/min/1.73 m2
Median hemoglobin [normal range 107 to 131 g/L]
74.25 g/L

iPatients can have multiple races selected.

CKD=chronic kidney disease; eGFR=estimated glomerular filtration rate; LDH=lactate dehydrogenase; U/L=units per liter.

Study endpoints2

Primary endpoint

Composite endpoint of complete TMA response:

  • Platelet count normalizationj
  • Serum LDH normalizationj
  • ≥25% improvement in serum creatinine from baseline

Patients had to meet all criteria for the primary endpoint of a complete TMA response at 2 separate assessments obtained at least 4 weeks (28 days) apart and any measurement in between.

jIncludes ≥150 x 109 cells/L for platelet count and ≤246 U/L for LDH.2


Select secondary endpoints

  • Time to complete TMA response and complete TMA response status over time
  • Dialysis requirement and CKD stage as evaluated by eGFR
  • Hemoglobin response
  • Change from baseline in quality of life

CKD=chronic kidney disease; eGFR=estimated glomerular filtration rate; LDH=lactate dehydrogenase; TMA=thrombotic microangiopathy; U/L=units per liter.

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Safety profile

Adverse reactions reported in ≥10% of ULTOMIRIS-treated pediatric patients with atypical-HUS at Week 261

Pediatric patients (N=16)
Body system Adverse reaction
All gradesk
(n=16)
n (%)
≥Grade 3
(n=6)
n (%)
Blood and lymphatic system disorders
Anemia
2 (13)
1 (6)
Lymphadenopathy
2 (13)
0 (0)
Gastrointestinal disorders
Diarrhea
6 (38)
0 (0)
Constipation
4 (25)
0 (0)
Vomiting
4 (25)
1 (6)
Abdominal pain
3 (19)
0 (0)
Nausea
2 (13)
0 (0)
General disorders and administration site conditions
Pyrexia
8 (50)
0 (0)
Infections and infestations
Upper respiratory tract infectionl
7 (44)
1 (6)
Gastroenteritis viral
2 (13)
2 (13)
Pneumonia
2 (13)
1 (6)
Tonsillitis
2 (13)
0 (0)
Injury, poisoning, and procedural complications
Confusion
3 (19)
0 (0)
Investigations
Vitamin D decreased
3 (19)
0 (0)
Metabolism and nutrition disorders
Decreased appetite
2 (13)
0 (0)
Iron deficiency
2 (13)
0 (0)
Musculoskeletal and connective tissue disorders
Myalgia
3 (19)
0 (0)
Pain in extremity
2 (13)
0 (0)
Nervous system disorders
Headache
5 (31)
0 (0)
Respiratory, thoracic, and mediastinal disorders
Cough
3 (19)
0 (0)
Dyspnea
2 (13)
0 (0)
Skin and subcutaneous tissue disorders
Rash
3 (19)
0 (0)
Vascular disorders
Hypertension
4 (25)
1 (6)
Hypotension
2 (13)
0 (0)
  • The most frequent adverse reactions reported in ≥20% of pediatric patients treated with ULTOMIRIS were upper respiratory tract infection, diarrhea, constipation, vomiting, headache, hypertension, and pyrexia.
  • Clinically relevant adverse reactions in <10% of patients included viral infection.
  • Serious adverse reactions were reported in 42 adult and pediatric patients (57%) with atypical-HUS receiving ULTOMIRIS. The most frequent serious adverse reactions reported in more than 2 patients (2.7%) treated with ULTOMIRIS were hypertension, pneumonia, and abdominal pain.

kGraded per CTCAE v5.0.

lGrouped term includes nasopharyngitis, pharyngitis, upper respiratory tract infection, rhinitis, viral upper respiratory tract infection, rhinovirus infection, viral pharyngitis, rhinorrhea, and oropharyngeal pain.

CTCAE=Common Terminology Criteria for Adverse Events.



Safety in pediatric patients at 50 weeks3,m

Event type
Overall (N=21)
n (%)
Events
Any AE
21 (100.0)
369
Any SAE
14 (66.7)
31
TEAE resulting in drug discontinuation
1 (4.8)
2
TESAE resulting in drug discontinuation
1 (4.8)
2
TEAE resulting in study discontinuation
1 (4.8)
2
TESAE resulting in study discontinuation
1 (4.8)
2
TEAE during study drug infusion
4 (19.0)
7
TESAE during study drug infusion
0 (0)
0
Treatment-related AEs
10 (47.6)
33
Meningococcal infections
0 (0)
0
Deaths
0 (0)
0

mSafety outcomes at the last available follow-up with ULTOMIRIS.

AE=adverse event; SAE=serious adverse event; TEAE=treatment-emergent adverse event; TESAE=treatment-emergent serious adverse event.



Most frequent treatment-emergent adverse events3

Adverse event
Overall (N=21)
n (%)
Events
Pyrexia
10 (47.6)
22
Nasopharyngitis
7 (33.3)
13
Diarrhea
7 (33.3)
10
Vomiting
7 (33.3)
25
Headache
7 (33.3)
19
Abdominal pain
6 (28.6)
11
Hypertension
6 (28.6)
8
Cough
5 (23.8)
6
Rash
4 (19.0)
5
Rhinorrhea
4 (19.0)
4
Myalgia
4 (19.0)
7
Constipation
4 (19.0)
8
Nausea
4 (19.0)
9

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This way to adult efficacy data

Read the efficacy and safety data for ULTOMIRIS studied in adult patients with atypical-HUS.

Explore adult data

Tools for the journey

Find additional resources and support information to help get your patients started on ULTOMIRIS.

View resources
References:
  1. ULTOMIRIS. Prescribing information. Alexion Pharmaceuticals, Inc.
  2. Data on file [ALXN1210-aHUS-312CSR].
  3. Ariceta G, et al. Kidney Int. 2021;100(1):225-237.
IMPORTANT SAFETY INFORMATION INCLUDING BOXED WARNING
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WARNING: SERIOUS MENINGOCOCCAL INFECTIONS

ULTOMIRIS, a complement inhibitor, increases the risk of serious infections caused by Neisseria meningitidis [see Warnings and Precautions (5.1)] Life-threatening and fatal meningococcal infections have occurred in patients treated with complement inhibitors. These infections may become rapidly life-threatening or fatal if not recognized and treated early.

  • Complete or update vaccination for meningococcal bacteria (for serogroups A, C, W, Y, and B) at least 2 weeks prior to the first dose of ULTOMIRIS, unless the risks of delaying ULTOMIRIS therapy outweigh the risk of developing a serious infection. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccinations against meningococcal bacteria in patients receiving a complement inhibitor. See Warnings and Precautions (5.1) for additional guidance on the management of the risk of serious infections caused by meningococcal bacteria.
  • Patients receiving ULTOMIRIS are at increased risk for invasive disease caused by Neisseria meningitidis, even if they develop antibodies following vaccination. Monitor patients for early signs and symptoms of serious meningococcal infections and evaluate immediately if infection is suspected.

Because of the risk of serious meningococcal infections, ULTOMIRIS is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called ULTOMIRIS and SOLIRIS REMS [see Warnings and Precautions (5.2)].

CONTRAINDICATIONS

  • Initiation in patients with unresolved serious Neisseria meningitidis infection.

WARNINGS AND PRECAUTIONS
Serious Meningococcal Infections
ULTOMIRIS, a complement inhibitor, increases a patient’s susceptibility to serious, life-threatening, or fatal infections caused by meningococcal bacteria (septicemia and/or meningitis) in any serogroup, including non-groupable strains. Life-threatening and fatal meningococcal infections have occurred in both vaccinated and unvaccinated patients treated with complement inhibitors.

Revaccinate patients in accordance with ACIP recommendations considering the duration of ULTOMIRIS therapy. Note that ACIP recommends an administration schedule in patients receiving complement inhibitors that differs from the administration schedule in the vaccine prescribing information. If urgent ULTOMIRIS therapy is indicated in a patient who is not up to date with meningococcal vaccines according to ACIP recommendations, provide antibacterial drug prophylaxis and administer meningococcal vaccines as soon as possible. Various durations and regimens of antibacterial drug prophylaxis have been considered, but the optimal durations and drug regimens for prophylaxis and their efficacy have not been studied in unvaccinated or vaccinated patients receiving complement inhibitors, including ULTOMIRIS. The benefits and risks of treatment with ULTOMIRIS, as well as those associated with antibacterial drug prophylaxis in unvaccinated or vaccinated patients, must be considered against the known risks for serious infections caused by Neisseria meningitidis.

Vaccination does not eliminate the risk of serious meningococcal infections, despite development of antibodies following vaccination.

Closely monitor patients for early signs and symptoms of meningococcal infection and evaluate patients immediately if infection is suspected. Inform patients of these signs and symptoms and instruct patients to seek immediate medical care if they occur. Promptly treat known infections. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early. Consider interruption of ULTOMIRIS in patients who are undergoing treatment for serious meningococcal infection depending on the risks of interrupting treatment in the disease being treated.

ULTOMIRIS and SOLIRIS REMS
Due to the risk of serious meningococcal infections, ULTOMIRIS is available only through a restricted program called ULTOMIRIS and SOLIRIS REMS.

Prescribers must enroll in the REMS, counsel patients about the risk of serious meningococcal infection, provide patients with the REMS educational materials, assess patient vaccination status for meningococcal vaccines (against serogroups A, C, W, Y, and B) and vaccinate if needed according to current ACIP recommendations two weeks prior to the first dose of ULTOMIRIS. Antibacterial drug prophylaxis must be prescribed if treatment must be started urgently, and the patient is not up to date with both meningococcal vaccines according to current ACIP recommendations at least two weeks prior to the first dose of ULTOMIRIS. Patients must receive counseling about the need to receive meningococcal vaccines and to take antibiotics as directed, signs and symptoms of meningococcal infection, and be instructed to carry the Patient Safety Card at all times during and for 8 months following ULTOMIRIS treatment.

Further information is available at www.UltSolREMS.com or 1-888-765-4747.

Other Infections
Serious infections with Neisseria species (other than Neisseria meningitidis), including disseminated gonococcal infections, have been reported.

ULTOMIRIS blocks terminal complement activation; therefore, patients may have increased susceptibility to infections, especially with encapsulated bacteria, such as infections caused by Neisseria meningitidis but also Streptococcus pneumoniae, Haemophilus influenzae, and to a lesser extent, Neisseria gonorrhoeae. Children treated with ULTOMIRIS may be at increased risk of developing serious infections due to Streptococcus pneumoniae and Haemophilus influenzae type b (Hib). Administer vaccinations for the prevention of Streptococcus pneumoniae and Haemophilus influenzae type b (Hib) infections according to ACIP recommendations. Patients receiving ULTOMIRIS are at increased risk for infections due to these organisms, even if they develop antibodies following vaccination.

Monitoring Disease Manifestations after ULTOMIRIS Discontinuation
 Treatment Discontinuation for PNH
After discontinuing treatment with ULTOMIRIS, closely monitor for signs and symptoms of hemolysis, identified by elevated LDH along with sudden decrease in PNH clone size or hemoglobin, or re-appearance of symptoms such as fatigue, hemoglobinuria, abdominal pain, shortness of breath (dyspnea), major adverse vascular event (including thrombosis), dysphagia, or erectile dysfunction. Monitor any patient who discontinues ULTOMIRIS for at least 16 weeks to detect hemolysis and other reactions. If signs and symptoms of hemolysis occur after discontinuation, including elevated LDH, consider restarting treatment with ULTOMIRIS.

Treatment Discontinuation for aHUS
ULTOMIRIS treatment of aHUS should be a minimum duration of 6 months. Due to heterogeneous nature of aHUS events and patient-specific risk factors, treatment duration beyond the initial 6 months should be individualized. There are no specific data on ULTOMIRIS discontinuation. After discontinuing treatment with ULTOMIRIS, patients should be monitored for clinical symptoms and laboratory signs of TMA complications for at least 12 months. TMA complications post-discontinuation can be identified if any of the following is observed: Clinical symptoms of TMA include changes in mental status, seizures, angina, dyspnea, thrombosis or increasing blood pressure. In addition, at least two of the following laboratory signs observed concurrently and results should be confirmed by a second measurement 28 days apart with no interruption: a decrease in platelet count of 25% or more as compared to either baseline or to peak platelet count during ULTOMIRIS treatment; an increase in serum creatinine of 25% or more as compared to baseline or to nadir during ULTOMIRIS treatment; or, an increase in serum LDH of 25% or more as compared to baseline or to nadir during ULTOMIRIS treatment. If TMA complications occur after discontinuation, consider reinitiation of ULTOMIRIS treatment or appropriate organ-specific supportive measures.

Thromboembolic Event Management
The effect of withdrawal of anticoagulant therapy during treatment with ULTOMIRIS has not been established. Treatment should not alter anticoagulant management.

Infusion-Related Reactions
Intravenous administration may result in systemic infusion-related reactions, including anaphylaxis and hypersensitivity reactions. In clinical trials, infusion-related reactions occurred in approximately 1 to 7% of patients treated with ULTOMIRIS. These events included lower back pain, drop in blood pressure, limb discomfort, drug hypersensitivity (allergic reaction), dysgeusia (bad taste), and drowsiness. These reactions did not require discontinuation of ULTOMIRIS. If signs of cardiovascular instability or respiratory compromise occur, interrupt ULTOMIRIS infusion and institute appropriate supportive measures.

ADVERSE REACTIONS
 Adverse Reactions for PNH
Adverse reactions reported in ≥10% or more of patients with PNH were upper respiratory tract infection and headache. Serious adverse reactions were reported in 15 (6.8%) patients receiving ULTOMIRIS. The serious adverse reactions in patients treated with ULTOMIRIS included hyperthermia and pyrexia. No serious adverse reaction was reported in more than 1 patient treated with ULTOMIRIS. One fatal case of sepsis was identified in a patient treated with ULTOMIRIS. In clinical studies, clinically relevant adverse reactions in 1% of adult patients include infusion-related reactions.

Adverse reactions reported in ≥10% of pediatric patients treated with ULTOMIRIS who were treatment-naïve vs. Eculizumab-experienced were anemia (20% vs. 25%), abdominal pain (0% vs. 38%), constipation (0% vs. 25%), pyrexia (20% vs. 13%), upper respiratory tract infection (20% vs. 75%), pain in extremity (0% vs. 25%), and headache (20% vs. 25%).

Adverse Reactions for aHUS
Most common adverse reactions in patients with aHUS (incidence ≥20%) were upper respiratory tract infection, diarrhea, nausea, vomiting, headache, hypertension and pyrexia. Serious adverse reactions were reported in 42 (57%) patients with aHUS receiving ULTOMIRIS. The most frequent serious adverse reactions reported in more than 2 patients (2.7%) treated with ULTOMIRIS were hypertension, pneumonia and abdominal pain.

Adverse reactions reported in ≥20% of pediatric patients treated with ULTOMIRIS were diarrhea, constipation, vomiting, pyrexia, upper respiratory tract infection, decreased vitamin D, headache, cough, rash, and hypertension.

Adverse Reactions for gMG
Most common adverse reactions in adult patients with gMG (incidence ≥10%) were diarrhea and upper respiratory tract infection. Serious adverse reactions were reported in 20 (23%) of patients treated with ULTOMIRIS and in 14 (16%) patients receiving placebo. The most frequent serious adverse reactions were infections reported in at least 8 (9%) patients treated with ULTOMIRIS and in 4 (4%) patients treated with placebo. Of these infections, one fatal case of COVID-19 pneumonia was identified in a patient treated with ULTOMIRIS and one case of infection led to discontinuation of ULTOMIRIS.

Adverse Reactions for NMOSD
Most common adverse reactions in adult patients with NMOSD (incidence ≥10%) were COVID-19, headache, back pain, arthralgia, and urinary tract infection. Serious adverse reactions were reported in 8 (13.8%) patients with NMOSD receiving ULTOMIRIS.

DRUG INTERACTIONS
 Plasma Exchange, Plasmapheresis, and Intravenous Immunoglobulins
Concomitant use of ULTOMIRIS with plasma exchange (PE), plasmapheresis (PP), or intravenous immunoglobulin (IVIg) treatment can reduce serum ravulizumab concentrations and requires a supplemental dose of ULTOMIRIS.

Neonatal Fc Receptor Blockers
Concomitant use of ULTOMIRIS with neonatal Fc receptor (FcRn) blockers (e.g., efgartigimod) may lower systemic exposures and reduce effectiveness of ULTOMIRIS. Closely monitor for reduced effectiveness of ULTOMIRIS.

INDICATIONS
Paroxysmal Nocturnal Hemoglobinuria (PNH)
ULTOMIRIS is indicated for the treatment of adult and pediatric patients one month of age and older with paroxysmal nocturnal hemoglobinuria (PNH).

Atypical Hemolytic Uremic Syndrome (aHUS)
ULTOMIRIS is indicated for the treatment of adult and pediatric patients one month of age and older with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA).

Limitation of Use:
ULTOMIRIS is not indicated for the treatment of patients with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS).

Generalized Myasthenia Gravis (gMG)
ULTOMIRIS is indicated for the treatment of adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive.

Neuromyelitis Optica Spectrum Disorder (NMOSD)
ULTOMIRIS is indicated for the treatment of adult patients with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody positive.

To report SUSPECTED ADVERSE REACTIONS, contact Alexion Pharmaceuticals, Inc. at 1-844-259-6783 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see full Prescribing Information for ULTOMIRIS, including Boxed WARNING regarding serious and life-threatening or fatal meningococcal infections.