- Nine abstracts accepted, including
long-term data and subgroup analyses from the Phase 3 PREVENT
clinical trial evaluating SOLIRIS® (eculizumab) for the treatment
of neuromyelitis optica spectrum disorder (NMOSD) -
- Breadth of data to be presented further
support the significant and sustained relapse reduction
demonstrated by SOLIRIS in the treatment of adults with NMOSD who
are anti-aquaporin-4 (AQP4) antibody-positive -
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that
nine abstracts have been accepted for presentation at the 72nd
annual meeting of the American Academy of Neurology (AAN) in
Toronto, Ontario, Canada from April 25 through May 1, 2020.
Long-term safety and efficacy results from the Phase 3 PREVENT
study and open-label extension of SOLIRIS® (eculizumab) in adults
with neuromyelitis optica spectrum disorder (NMOSD) who are
anti-aquaporin-4 (AQP4) antibody-positive will be presented,
demonstrating the percentage of SOLIRIS patients who were
relapse-free remained high (94 percent) through 192 weeks.
Additional PREVENT data to be presented highlight the efficacy of
SOLIRIS and its impact on health outcomes, hospitalization rates
and relapse treatment, disability worsening and quality of life,
and the evidence of benefit of SOLIRIS across a broad range of
patients with NMOSD.
The accepted abstracts are listed below and are now available on
the AAN website:
Oral Presentations
Long-Term Safety and Efficacy of Eculizumab in Neuromyelitis
Optica Spectrum Disorder. Oral presentation during session S34:
Clinical Trials and Therapeutics in Autoimmune Neurology, April 28,
2020, 4:30 p.m. ET.
Efficacy and Safety of Eculizumab in Patients with Neuromyelitis
Optica Spectrum Disorder Previously Treated with Rituximab:
Findings from the Phase 3 PREVENT Study. Oral presentation during
session S34: Clinical Trials and Therapeutics in Autoimmune
Neurology, April 28, 2020, 4:18 p.m. ET.
Poster Presentations
Impact of Eculizumab on Health Outcomes in Patients with
Aquaporin-4 Antibody-Positive Neuromyelitis Optica Spectrum
Disorder: Findings from the PREVENT Study. Poster presentation
during session P14: Autoimmune Neurology: NMOSD and MOG 2, April
30, 2020, 8:00 – 9:00 a.m. ET and session P17: Neuroinflammation,
May 1, 2020, 11:30 a.m. – 1:00 p.m. ET.
Impact of Eculizumab on Hospitalization Rates and Relapse
Treatment in Patients with Aquaporin-4 Antibody-Positive
Neuromyelitis Optica Spectrum Disorder: the Phase 3 PREVENT Study.
Poster presentation during session P14: Autoimmune Neurology: NMOSD
and MOG 2, April 30, 2020, 8:00 – 9:00 a.m. ET and session P17:
Neuroinflammation, May 1, 2020, 11:30 a.m. – 1:00 p.m. ET.
Impact of Eculizumab on Disability Worsening and Quality of Life
in Patients with Aquaporin-4 Antibody-Positive Neuromyelitis Optica
Spectrum Disorder: Results from the Phase 3 PREVENT Study. Poster
presentation during session P14: Autoimmune Neurology: NMOSD and
MOG 2, April 30, 2020, 8:00 – 9:00 a.m. ET and session P17:
Neuroinflammation, May 1, 2020, 11:30 a.m. – 1:00 p.m. ET.
Infection Risk in Patients with Complement-Mediated Neurological
Disorders Receiving Eculizumab: Findings from Two Phase 3 Studies
and Their Extensions in Aquaporin-4 Antibody-Positive Neuromyelitis
Optica Spectrum Disorder (AQP4+ NMOSD) and Acetylcholine-Receptor
Antibody-Positive Refractory Generalized Myasthenia Gravis (AChR+
gMG). Poster presentation during session P14: Autoimmune Neurology:
NMOSD and MOG 2, April 30, 2020, 8:00 – 9:00 a.m. ET.
The Impact of Relapses on Quality of Life in Patients with
Neuromyelitis Optica Spectrum Disorder: Data from the Phase 3
PREVENT Study. Poster presentation during session P14: Autoimmune
Neurology: NMOSD and MOG 2, April 30, 2020, 8:00 – 9:00 a.m.
ET.
Benefit of Eculizumab for a Broad Range of Patients with
Aquaporin-4 Antibody-Positive Neuromyelitis Optica Spectrum
Disorder: Findings from the Phase 3 PREVENT Study. Poster
presentation during session P14: Autoimmune Neurology: NMOSD and
MOG 2, April 30, 2020, 8:00 – 9:00 a.m. ET and session P17:
Neuroinflammation, May 1, 2020, 11:30 a.m. – 1:00 p.m. ET.
Neuromyelitis Optica Spectrum Disorder (NMOSD): Epidemiology,
Treatments, and Outcomes in a Single Center. Poster presentation
during session P14: Autoimmune Neurology: NMOSD and MOG 2, April
30, 2020, 8:00 – 9:00 a.m. ET.
About Neuromyelitis Optica Spectrum Disorder (NMOSD)
Neuromyelitis Optica Spectrum Disorder (NMOSD) is a rare autoimmune
disease of the central nervous system (CNS). Approximately
three-quarters of NMOSD patients have anti-AQP4 antibody-positive
NMOSD. In patients with these antibodies, NMOSD occurs when the
complement system—a part of the body’s immune
system—over-responds—leading the body to primarily attack the optic
nerves and/or spinal cord in the CNS. People living with NMOSD
often experience unpredictable attacks, also referred to as
relapses, which tend to be severe and recurrent and may result in
permanent disability. The most common symptoms of NMOSD are optic
neuritis, which can cause visual problems including blindness, and
transverse myelitis, which can cause mobility problems including
paralysis. The disease primarily affects women, with an average age
of onset of 39 years. NMOSD is more common and more severe in
non-Caucasian populations worldwide.
About Generalized Myasthenia Gravis (gMG) Myasthenia
gravis (MG) is a rare, progressive, autoimmune neuromuscular
disease. In patients with anti-acetylcholine receptor (AchR)
antibody-positive MG, the body’s own immune system over-responds,
leading the body to attack its own healthy cells and produce
antibodies to fight against AchR, a receptor located on muscle
cells at the neuromuscular junction. As a result, communication
between the nerves and muscles is impaired, leading to a loss of
normal muscle function. MG typically begins with weakness in the
muscles that control the movements of the eyes and eyelids and
often progresses to the more severe and generalized form, known as
generalized myasthenia gravis (gMG). People with gMG can suffer
from slurred speech, choking, difficulty swallowing, drooping of
the eyelids, double or blurred vision, disabling fatigue,
immobility requiring assistance, shortness of breath and episodes
of respiratory failure that can be life-threatening. Complications,
exacerbations and myasthenic crises can require hospital and
intensive care unit admissions with prolonged stays. gMG can occur
at any age but most commonly begins before the age of 40 in women
and after the age of 60 in men.
About SOLIRIS® (eculizumab) SOLIRIS® (eculizumab) is a
first-in-class C5 complement inhibitor. 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 terminal complement cascade over-responds, leading the
body to attack its own healthy cells. SOLIRIS is administered
intravenously every two weeks, following an introductory dosing
period. In many countries around the world, SOLIRIS is approved to
treat paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic
uremic syndrome (aHUS), adults with generalized myasthenia gravis
(gMG) who are acetylcholine receptor (AchR) antibody positive
and/or adults with neuromyelitis optica spectrum disorder (NMOSD)
who are anti-aquaporin-4 (AQP4) antibody positive. SOLIRIS is not
indicated for the treatment of patients with Shiga-toxin E.
coli-related hemolytic uremic syndrome (STEC-HUS). To learn more
about the regulatory status of SOLIRIS in the countries that we
serve, please visit www.alexion.com.
INDICATIONS & IMPORTANT SAFETY INFORMATION FOR SOLIRIS®
(eculizumab) 300 mg / 30 mL injection for intravenous use
U.S. INDICATIONS
What is SOLIRIS? SOLIRIS is a prescription medicine
called a monoclonal antibody. SOLIRIS is used to treat patients
with a disease called Paroxysmal Nocturnal Hemoglobinuria (PNH).
SOLIRIS is used to treat adults and children with a disease called
atypical Hemolytic Uremic Syndrome (aHUS). SOLIRIS is not for use
in treating people with Shiga toxin E. coli related hemolytic
uremic syndrome (STEC-HUS). SOLIRIS is used to treat adults with a
disease called generalized myasthenia gravis (gMG) who are
anti-acetylcholine receptor (AchR) antibody-positive. SOLIRIS is
used to treat adults with a disease called neuromyelitis optica
spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4)
antibody-positive. It is not known if SOLIRIS is safe and effective
in children with PNH, gMG, or NMOSD.
U.S. IMPORTANT SAFETY INFORMATION SOLIRIS is a medicine
that affects the immune system. SOLIRIS can lower the ability of
the immune system to fight infections. SOLIRIS increases the chance
of getting serious and life-threatening meningococcal infections.
Meningococcal infections may quickly become life-threatening and
cause death if not recognized and treated early.
Meningococcal vaccines must be received at least two weeks
before the first dose of SOLIRIS if one has not already had this
vaccine. If one’s doctor decided that urgent treatment with SOLIRIS
is needed, meningococcal vaccination should be administered as soon
as possible. If one has not been vaccinated and SOLIRIS therapy
must be initiated immediately, two weeks of antibiotics should also
be administered with the vaccinations. If one had a meningococcal
vaccine in the past, additional vaccination might be needed before
starting SOLIRIS. Patients should ask their doctor if an additional
meningococcal vaccination is needed. Meningococcal vaccines reduce
the risk of meningococcal infection but do not prevent all
meningococcal infections. Call one’s doctor or get emergency
medical care right away if any of these signs and symptoms of a
meningococcal infection occur: 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.
One’s doctor will provide a Patient Safety Card about the risk
of meningococcal infection. Carry the card at all times during
treatment and for 3 months after the last SOLIRIS dose.
SOLIRIS is only available through a program called the
SOLIRIS REMS.
SOLIRIS may also increase the risk of other types of serious
infections. If one’s child is treated with SOLIRIS, make sure that
the child receives vaccinations against Streptococcus pneumoniae
and Haemophilus influenzae type b (Hib). Certain people may be at
risk of serious infections with gonorrhea. Talk to the doctor about
whether one is at risk for gonorrhea infection, about gonorrhea
prevention, and regular testing. Certain fungal infections
(Aspergillus) may also happen if one takes SOLIRIS and has a weak
immune system or a low white blood cell count.
Do not receive SOLIRIS if one has a meningococcal infection, or
has not been vaccinated against meningitis infection unless one’s
doctor decides that urgent treatment with SOLIRIS is needed.
Before one receives SOLIRIS, tell the doctor about all of the
medical conditions, including if one: has an infection or fever, is
pregnant or plans to become pregnant, and is breastfeeding or plans
to breastfeed. It is not known if SOLIRIS will harm an unborn baby
or if SOLIRIS passes into the breast milk.
Tell the doctor about all the medicines one takes, including
prescription and over-the-counter medicines, vitamins, and herbal
supplements. SOLIRIS and other medicines can affect each other,
causing side effects.
It is important that one: has all recommended vaccinations
before starting SOLIRIS, receives 2 weeks of antibiotics if one
immediately starts SOLIRIS, and stays up-to-date with all
recommended vaccinations during treatment with SOLIRIS. Know the
medications one takes and the vaccines one receives. Keep a list of
them to show the doctor and pharmacist when one gets a new
medicine.
If one has PNH, the doctor will need to monitor closely for at
least 8 weeks after stopping SOLIRIS. Stopping treatment with
SOLIRIS may cause breakdown of the red blood cells due to PNH.
Symptoms or problems that can happen due to red blood cell
breakdown include: drop in the number of the red blood cell count,
drop in the platelet counts, confusion, kidney problems, blood
clots, difficulty breathing, and chest pain. If one has aHUS, the
doctor will need to monitor closely during and for at least 12
weeks after stopping treatment for signs of worsening aHUS symptoms
or problems related to abnormal clotting (thrombotic
microangiopathy). Symptoms or problems that can happen with
abnormal clotting may include: stroke, confusion, seizure, chest
pain (angina), difficulty breathing, kidney problems, swellings in
arms or legs, and a drop in the platelet count.
SOLIRIS can cause serious side effects including serious
allergic reactions. Serious allergic reactions can happen during
one’s SOLIRIS infusion. Tell the doctor or nurse right away if one
gets any of these symptoms during the SOLIRIS infusion: chest pain,
trouble breathing or shortness of breath, swelling of the face,
tongue, or throat, and feeling faint or pass out. If one has an
allergic reaction to SOLIRIS, the doctor may need to infuse SOLIRIS
more slowly, or stop SOLIRIS.
The most common side effects in people with PNH treated with
SOLIRIS include: headache, pain or swelling of the nose or throat
(nasopharyngitis), back pain, and nausea. The most common side
effects in people with aHUS treated with SOLIRIS include: headache,
diarrhea, high blood pressure (hypertension), common cold (upper
respiratory infection), stomach-area (abdominal) pain, vomiting,
pain or swelling of the nose or throat (nasopharyngitis), low red
blood cell count (anemia), cough, swelling of legs or feet
(peripheral edema), nausea, urinary tract infections, and fever.
The most common side effects in people with gMG treated with
SOLIRIS include: muscle and joint (musculoskeletal) pain. The most
common side effects in people with NMOSD treated with SOLIRIS
include: common cold (upper respiratory infection); pain or
swelling of the nose or throat (nasopharyngitis); diarrhea; back
pain; dizziness; flu like symptoms (influenza) including fever,
headache, tiredness, cough, sore throat, and body aches; joint pain
(arthralgia); throat irritation (pharyngitis), and bruising
(contusion).
Please see the accompanying full Prescribing Information and
Medication Guide for SOLIRIS, including BOXED WARNING regarding
serious and life-threatening meningococcal infections.
About Alexion Alexion is a global biopharmaceutical
company focused on serving patients and families affected by rare
diseases through the discovery, development and commercialization
of life-changing medicines. As the global leader in complement
biology and inhibition for more than 20 years, Alexion has
developed and commercializes two approved complement inhibitors to
treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and
atypical hemolytic uremic syndrome (aHUS), as well as the first and
only approved complement inhibitor to treat anti-acetylcholine
receptor (AchR) antibody-positive generalized myasthenia gravis
(gMG) and neuromyelitis optica spectrum disorder (NMOSD). Alexion
also has two highly innovative enzyme replacement therapies for
patients with life-threatening and ultra-rare metabolic disorders,
hypophosphatasia (HPP) and lysosomal acid lipase deficiency
(LAL-D). In addition, the company is developing several
mid-to-late-stage therapies, including a copper-binding agent for
Wilson disease, an anti-neonatal Fc receptor (FcRn) antibody for
rare Immunoglobulin G (IgG)-mediated diseases and an oral Factor D
inhibitor as well as several early-stage therapies, including one
for light chain (AL) amyloidosis, a second oral Factor D inhibitor
and a third complement inhibitor. Alexion focuses its research
efforts on novel molecules and targets in the complement cascade
and its development efforts on the core therapeutic areas of
hematology, nephrology, neurology, metabolic disorders and
cardiology. Headquartered in Boston, Massachusetts, Alexion has
offices around the globe and serves patients in more than 50
countries. This press release and further information about Alexion
can be found at: www.alexion.com.
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Media Megan Goulart, 857-338-8634 Senior Director,
Corporate Communications
Investors Susan Altschuller, Ph.D., 857-338-8788 Vice
President, Investor Relations
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