Zero adjudicated relapses observed among
ULTOMIRIS patients over a median treatment duration of 73
weeks
Positive high-level results from the open-label Phase III
CHAMPION-NMOSD trial showed that ULTOMIRIS® (ravulizumab-cwvz)
achieved a statistically significant and clinically meaningful
reduction in the risk of relapse in adults with anti-aquaporin-4
(AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum
disorder (NMOSD) compared to the external placebo arm from the
pivotal SOLIRIS® PREVENT clinical trial.
ULTOMIRIS, the first and only long-acting C5 complement
inhibitor, met the primary endpoint of time to first on-trial
relapse, as confirmed by an independent adjudication committee.
Notably, no relapse was observed in 58 patients over a median
treatment duration of 73 weeks.
NMOSD is a rare and devastating autoimmune disease that affects
the central nervous system (CNS), including the spine and optic
nerves.1-3 Most people living with NMOSD often experience
unpredictable relapses, a new onset of neurologic symptoms or
worsening of existing neurologic symptoms, also referred to as
attacks, which tend to be severe and recurrent and may result in
permanent disability.4-6
Sean J. Pittock, MD, Director of Mayo Clinic's Center for
Multiple Sclerosis and Autoimmune Neurology and of Mayo's
Neuroimmunology Laboratory and lead primary investigator in the
CHAMPION-NMOSD trial, said: “Every NMOSD relapse can have
debilitating and irreversible consequences, so reducing relapses is
critical. Patients on ULTOMIRIS remained relapse free over a median
treatment duration of 73 weeks in the trial.”
Marc Dunoyer, Chief Executive Officer, Alexion, said: “SOLIRIS
established the role of complement inhibition in preventing
relapses in NMOSD, and with ULTOMIRIS, we continue to innovate for
patients with a more convenient every eight-week dosing schedule.
These trial results show that ULTOMIRIS may help patients move
towards eliminating relapses, which is an important advancement in
the treatment of NMOSD.”
The safety and tolerability of ULTOMIRIS in the Champion-NMOSD
trial were consistent with previous clinical studies and other
approved indications. Fifty-six patients are continuing to receive
treatment in a long-term extension period, which is ongoing.
The data will be presented at a forthcoming medical meeting and
submitted to global health authorities as rapidly as possible to
bring forward ULTOMIRIS to the NMOSD community.
INDICATION(S) & IMPORTANT SAFETY INFORMATION for
ULTOMIRIS® (ravulizumab-cwvz)
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).
- adults and children 1 month of age and older with a disease
called atypical Hemolytic Uremic Syndrome (aHUS). ULTOMIRIS is not
used in treating people with Shiga toxin E. coli related hemolytic
uremic syndrome (STEC-HUS).
- adults with a disease called generalized myasthenia gravis
(gMG) who are anti-acetylcholine receptor (AChR) antibody
positive.
It is not known if ULTOMIRIS is safe and effective in children
younger than 1 month of age.
IMPORTANT SAFETY INFORMATION 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.
- You must receive meningococcal vaccines at least 2 weeks before
your first dose of ULTOMIRIS if you are not vaccinated.
- If your doctor decided that urgent treatment with ULTOMIRIS is
needed, you should receive meningococcal vaccination as soon as
possible.
- If you have not been vaccinated and ULTOMIRIS therapy must be
initiated immediately, you should also receive 2 weeks of
antibiotics with your vaccinations.
- If you had a meningococcal vaccine in the past, you might need
additional vaccination. Your doctor will decide if you need
additional vaccination.
- 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 Haemophilis 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.
If you have aHUS, your doctor will need to monitor you
closely for at least 12 months after stopping treatment for signs
of worsening aHUS or problems related to a type of abnormal
clotting and breakdown of your red blood cells called thrombotic
microangiopathy (TMA). Symptoms or problems that can happen with
TMA may include: confusion or loss of consciousness, seizures,
chest pain (angina), difficulty breathing and blood clots or
stroke.
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, tiredness, feeling faint,
discomfort in your arms or legs, or bad taste. 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.
The most common side effects of ULTOMIRIS in people with aHUS
are upper respiratory tract infection, diarrhea, nausea, vomiting,
headache, high blood pressure and fever.
The most common side effects of ULTOMIRIS in people with gMG
are diarrhea and upper respiratory tract infection.
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.
Please see the accompanying full Prescribing Information and
Medication Guide for ULTOMIRIS, including Boxed WARNING regarding
serious and life-threatening meningococcal
infections/sepsis.
Notes
NMOSD NMOSD is a rare disease in which the immune system
is inappropriately activated to target healthy tissues and cells in
the CNS.1,2 Approximately three-quarters of people with NMOSD are
anti-AQP4 Ab+, meaning they produce antibodies that bind to a
specific protein, aquaporin-4 (AQP4).7 This binding can
inappropriately activate the complement system, which is part of
the immune system and is essential to the body’s defense against
infection, to destroy cells in the optic nerve, spinal cord and
brain.1,8,9
It most commonly affects women and begins in the mid-30s. Men
and children may also develop NMOSD, but it is even more rare.10,11
People with NMOSD may experience vision problems, intense pain,
loss of bladder/bowel function, abnormal skin sensations (eg,
tingling, prickling or sensitivity to heat/cold) and impact on
coordination and/or movement.3-5,12,13 Most people living with
NMOSD experience unpredictable relapses, also known as attacks.
Each relapse can result in cumulative disability including vision
loss, paralysis and sometimes premature death.4-6 NMOSD is a
distinct disease from other CNS diseases, including multiple
sclerosis. The journey to diagnosis can be long, with the disease
sometimes misdiagnosed.14-16
CHAMPION-NMOSD CHAMPION-NMOSD is a global Phase III,
open-label, multicenter trial evaluating the safety and efficacy of
ULTOMIRIS in adults with NMOSD. The trial enrolled 58 patients
across North America, Europe, Asia-Pacific and Japan. Participants
were required to have a confirmed NMOSD diagnosis with a positive
anti-AQP4 antibody test, at least one attack or relapse in the
twelve months prior to the screening visit, an Expanded Disability
Status Scale Score of 7 or less and body weight of at least 40
kilograms at trial entry. Participants could stay on stable
supportive immunosuppressive therapy for the duration of the
trial.17
Due to the potential long-term functional impact of NMOSD
relapses, a direct placebo comparator arm was precluded for ethical
reasons. The active treatment was compared to an external placebo
arm from the pivotal SOLIRIS PREVENT clinical trial.
Over a median treatment duration of 73 weeks, all enrolled
patients received a single weight-based loading dose of ULTOMIRIS
on Day 1, followed by regular weight-based maintenance dosing
beginning on Day 15, every eight weeks. The primary endpoint was
time to first on-trial relapse, as confirmed by an independent
adjudication committee. The end of the primary treatment period
could have occurred either when all patients completed or
discontinued prior to the Week 26 visit and two or more adjudicated
relapses were observed, or when all patients completed or
discontinued prior to the Week 50 visit if fewer than two
adjudicated relapses were observed. In the trial, there were zero
adjudicated relapses so the end of the primary treatment period
occurred when the last enrolled participant completed the 50 week
visit.
Patients who completed the primary treatment period were
eligible to continue into a long-term extension period, which is
ongoing.
ULTOMIRIS ULTOMIRIS (ravulizumab-cwvz), the first and
only long-acting C5 complement inhibitor, offers immediate,
complete and sustained complement inhibition. The medication works
by inhibiting the C5 protein in the terminal complement cascade, a
part of the body’s immune system. When activated in an uncontrolled
manner, the complement cascade over-responds, leading the body to
attack its own healthy cells. ULTOMIRIS is administered
intravenously every eight weeks in adult patients, following a
loading dose.
ULTOMIRIS is approved in the US for the treatment of certain
adults with generalized myasthenia gravis.
ULTOMIRIS is also approved in the US, EU and Japan for the
treatment of certain adults and children with paroxysmal nocturnal
hemoglobinuria.
Additionally, ULTOMIRIS is approved in the US, EU and Japan for
certain adults and children with atypical hemolytic uremic syndrome
to inhibit complement-mediated thrombotic microangiopathy.
As part of a broad development program, ULTOMIRIS is being
assessed for the treatment of additional hematology and neurology
indications.
Alexion Alexion, AstraZeneca Rare Disease, is the group
within AstraZeneca focused on rare diseases, created following the
2021 acquisition of Alexion Pharmaceuticals, Inc. As a leader in
rare diseases for nearly 30 years, Alexion is focused on serving
patients and families affected by rare diseases and devastating
conditions through the discovery, development and commercialization
of life-changing medicines. Alexion focuses its research efforts on
novel molecules and targets in the complement cascade and its
development efforts on hematology, nephrology, neurology, metabolic
disorders, cardiology and ophthalmology. Headquartered in Boston,
Massachusetts, Alexion has offices around the globe and serves
patients in more than 50 countries. For more information, please
visit www.alexion.com.
About AstraZeneca AstraZeneca is a global, science-led
biopharmaceutical company that focuses on the discovery,
development and commercialization of prescription medicines in
Oncology, Rare Diseases and BioPharmaceuticals, including
Cardiovascular, Renal & Metabolism, and Respiratory &
Immunology. Based in Cambridge, UK, AstraZeneca operates in over
100 countries and its innovative medicines are used by millions of
patients worldwide. For more information, please visit
www.astrazeneca-us.com and follow us on Twitter @AstraZenecaUS.
References
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Weinshenker BG. The spectrum of neuromyelitis optica. Lancet
Neurol. 2007;6(9):805-815.
- Wingerchuk DM. Diagnosis and treatment of neuromyelitis optica.
Neurologist. 2007;13(1):2-11.
- Hamid SHM, Whittam D, Mutch K et al. What proportion of
AQP4-IgG-negative NMO spectrum disorder patients are MOG-IgG
positive? A cross sectional study of 132 patients. J Neurol.
2017;264(10):2088-2094.
- Wingerchuk DM, Weinshenker BG. Neuromyelitis optica. Curr Treat
Options Neurol. 2008;10(1):55-66.
- Kitley J, Leite MI, Nakashima I, et al. Prognostic factors and
disease course in aquaporin-4 antibody-positive patients with
neuromyelitis optica spectrum disorder from the United Kingdom and
Japan. Brain. 2012;135(6):1834-1849.
- Jarius S, Ruprecht K, Wildemann B, et al. Contrasting disease
patterns in seropositive and seronegative neuromyelitis optica: a
multicentre study of 175 patients. J Neuroinflamm. 2012;9:14.
- Wingerchuk DM, Hogancamp WF, O’Brien PC, Weinshenker BG. The
clinical course of neuromyelitis optica (Devic’s syndrome).
Neurology. 1999;53(5):1107-1114.
- Papadopoulos MC, Bennett JL, Verkman AS. Treatment of
neuromyelitis optica: state-of-the-art and emerging therapies. Nat
Rev Neurol. 2014;10(9):493.
- Cossburn, M., et al. (2012). The Prevalence of Neuromyelitis
Optica in South East Wales." Eur J Neurol., 19(4): 655-659.
- Takata K, Matsuzaki T, Tajika Y. Aquaporins: water channel
proteins of the cell membrane. Prog Histochem Cytochem.
2004;39(1):1-83.
- Mori M, Kuwabara S, Paul F. Worldwide prevalence of
neuromyelitis optica spectrum disorders. J Neurol Neurosurg
Psychiatry. 2018 Jun;89(6):555-556. doi: 10.1136/jnnp-2017-317566.
Epub 2018 Feb 7. PMID: 29436488.
- Quek AML, Mckeon A, Lennon VA et al. Effects of age and sex on
aquaporin-4 autoimmunity. Arch Neurol 2012 and 69:1039–43.
- Tüzün E, Kürtüncü M, Türkoğlu R, et al. Enhanced complement
consumption in neuromyelitis optica and Behcet’s disease patients.
J Neuroimmunol. 2011;233(1-2):211-215.
- Kuroda H, Fujihara K, Takano R, et al. Increase of complement
fragment C5a in cerebrospinal fluid during exacerbation of
neuromyelitis optica. J Neuroimmunol. 2013;254(1-2):178-182.
- Jarius, S., Wildemann, B. (2013). The History of Neuromyelitis
Optica. J Neuroinflammation 10, 797.
- Mealy, M. A., et al. (2019). Assessment of Patients with
Neuromyelitis Optica Spectrum Disorder Using the EQ-5D.
International journal of MS care, 21(3), 129–134.
- ClinicalTrials.gov. An Efficacy and Safety Study of Ravulizumab
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Available online. Accessed April 2022.
Dr. Pittock has provided consulting services to Alexion.
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