ajor adverse vascular event
Efficacy was established based on hemolysis as measured by LDH percent change frombaseline to Day 183 and supportive efficacy data was transfusion avoidance, proportionof patients with stabilized hemoglobin, and the proportion of patients with breakthroughhemolysis through Day 183.
Non-inferiority of ULTOMIRIS to eculizumab was demonstrated across endpoints in thepatients with PNH previously treated with eculizumab described in the table below.
Table 9:
Efficacy Results in the Eculizumab-Experienced Patients with PNH
Eculizumab-Experienced Study
ULTOMIRIS Eculizumab Statistic for Treatment
n = 97 n = 98 Comparison Effect
(95% CI)
LDH Percent change -0.82% 8.4% Difference 9.2
in % change (-0.42, 18.8)
from
baseline
Breakthrough hemolysis 0% 5.1% Difference
in rate
5.1
(-8.9, 19.0)
Transfusion avoidance 87.6 % 82.7% Difference
in rate
5.5
(-4.3, 15.7)
Hemoglobin
Stabilization
76.3% 75.5% Difference
in rate
1.4
(-10.4, 13.3)
Note: CI = confidence interval
There was no observable difference in fatigue between ULTOMIRIS and eculizumabafter 26 weeks of treatment compared to baseline as measured by the FACIT-fatigue
instrument. Patient-reported fatigue may be an under-or over-estimation, becausepatients were not blinded to treatment assignment.
16 HOW SUPPLIED/STORAGE AND HANDLING
ULTOMIRIS (ravulizumab-cwvz) injection is a clear to translucent, slight whitish colorpreservative-free, solution supplied as one 300 mg/30 mL (10 mg/mL) single-dose vialper carton. NDC 25682-022-01.
Store ULTOMIRIS vials refrigerated at 2°C – 8°C (36°F – 46°F) in the original carton to
protect from light. Do not freeze. Do not shake.
Refer to Dosage and Administration (2.3) for information on the stability and storage ofdiluted solutions of ULTOMIRIS.
17 PATIENT COUNSELING INFORMATION
Advise the patient to read FDA-approved patient labeling (Medication Guide).
Meningococcal Infection
Advise patients of the risk of meningococcal infection/sepsis. Inform patients that theyare required to receive meningococcal vaccination at least 2 weeks prior to receiving thefirst dose of ULTOMIRIS, if they have not previously been vaccinated.
They arerequired to be revaccinated according to current medical guidelines for meningococcalvaccines use while on ULTOMIRIS therapy. Inform patients that vaccination may notprevent meningococcal infection. Inform patients about the signs and symptoms ofmeningococcal infection/sepsis, and strongly advise patients to seek immediate medicalattention if these signs or symptoms occur. These signs and symptoms are as follows:
• headache with nausea or vomiting
• headache and a 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
Inform patients that they will be given an ULTOMIRIS Patient Safety Card that theyshould carry with them at all times. This card describes symptoms which, if experienced,should prompt the patient to immediately seek medical eva luation.
Other Infections
Counsel patients of the increased risk of infections, particularly those due to encapsulatedbacteria, especially Neisseria species. Advise patients of the need for vaccination againstmening |
