- SOLIRIS® (eculizumab) is the first and only
approved medication for NMOSD in Japan -
- 98% of adult anti-aquaporin-4 (AQP4)
antibody-positive patients treated with SOLIRIS were relapse free
compared to 63% receiving placebo at 48 weeks -
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that
Japan’s Ministry of Health, Labour and Welfare (MHLW) has approved
the extension of the current marketing authorization of SOLIRIS®
(eculizumab) to include the prevention of relapse in patients with
anti-aquaporin-4 (AQP4) antibody-positive neuromyelitis optica
spectrum disorder (NMOSD), including neuromyelitis optica.
“We are pleased that the Japanese health authorities have
approved SOLIRIS as a new treatment for patients suffering from
this complex and unpredictable disease,” said John Orloff, M.D.,
Executive Vice President and Head of Research and Development at
Alexion. “Nearly all patients treated with SOLIRIS were relapse
free at 48 weeks in the Phase 3 PREVENT study, providing new hope
for Japanese patients with NMOSD and their clinicians.”
NMOSD is a rare, devastating, complement-mediated disorder of
the central nervous system characterized by relapses, also referred
to as attacks. Each attack can result in stepwise accumulation of
disability, including blindness and paralysis and sometimes
premature death. NMOSD disproportionately strikes young women in
the prime of their lives, with the average age of first onset at
just 39 years. Previously known as Devic’s Disease, NMOSD is often
confused with other neurological illnesses such as multiple
sclerosis (MS), which can lead to delays in diagnosis and treatment
with medicines that can worsen disease progression.
“The understanding of NMOSD has rapidly evolved in recent years
since complement activation by AQP4 antibodies was identified as an
underlying cause of the disease,” said Kazuo Fujihara, Professor,
Fukushima Medical University, Director of the Multiple Sclerosis
& Neuromyelitis Optica Center at Southern Tohoku Research
Institute and a principal investigator in the PREVENT trial of
SOLIRIS in anti-AQP4 antibody-positive NMOSD. “With the first
approved medicine for NMOSD in Japan, SOLIRIS will provide highly
effective treatment to prevent future relapses in these
patients.”
The approval of SOLIRIS was based on comprehensive results from
the Phase 3 randomized, double-blind placebo controlled PREVENT
trial, which were published in The New England Journal of Medicine
and a long-term extension study (ECU-NMO-302), which is still
underway. In the PREVENT study, patients with NMOSD who were
anti-AQP4 antibody-positive were treated with SOLIRIS (n=96) or
placebo (n=47). The study met its primary endpoint of prolonging
the time to first adjudicated relapse and reducing the risk of
relapse. At 48 weeks, 98 percent of patients treated with SOLIRIS
were relapse free compared to 63 percent of patients receiving
placebo. Of the approximately one quarter of patients treated
solely with SOLIRIS monotherapy, without receiving other
immunosuppressive therapies, 100 percent were relapse free at 48
weeks compared to 61 percent in the placebo group. Sustained
effects of SOLIRIS were observed through 144 weeks of
treatment.
The safety profile of SOLIRIS was consistent with that seen for
SOLIRIS in other clinical studies and real-world use in its three
approved indications. The most common adverse events observed in
the PREVENT study were upper respiratory tract infection (29
percent of patients in the SOLIRIS group vs. 13 percent in the
placebo group), headache (23 vs. 23 percent), nasopharyngitis (21
vs. 19 percent) and nausea (17 vs. 26 percent). The serious adverse
events that were reported for more than one patient in either group
were pneumonia (three patients in the SOLIRIS group vs. one patient
in the placebo group) and cellulitis, sepsis and urinary tract
infection (two patients for each event in the SOLIRIS group vs. no
patient in the placebo group). One patient receiving SOLIRIS and
concomitant supportive IST died from a pulmonary empyema. The
patient had an extensive history of pulmonary disease and was an
active smoker. No cases of meningococcal infection were observed in
the study.
SOLIRIS was approved for the treatment of NMOSD in adult
patients who are anti-AQP4 antibody-positive by the U.S. Food and
Drug Administration (FDA) in June 2019 and by the European
Commission (EC) in August 2019. SOLIRIS received Orphan Drug
Designation (ODD) for the treatment of NMOSD in the U.S., EU and
Japan.
About NMOSD NMOSD is a rare and severe, autoimmune,
inflammatory disorder that attacks the central nervous system
(CNS), in which complement activation due to anti-aquaporin-4
(AQP4) antibodies plays a significant role in the disease process.
Patients with NMOSD experience unpredictable attacks, also referred
to as relapses, which can cause irreversible damage to the optic
nerve and spinal cord and can lead to long-term disability. The
most common symptoms of NMOSD are optic neuritis and transverse
myelitis. Optic neuritis can cause visual problems including
blindness; transverse myelitis can cause mobility problems
including paralysis. The disease primarily affects women, often in
the prime of their lives, with an average age of onset of 39 years.
The prevalence of NMOSD may be more common and more severe in
non-Caucasian populations worldwide.
Approximately three quarters (73%) of all patients with NMOSD
have AQP4 auto-antibodies. In patients with anti-AQP4
antibody-positive NMOSD, the body’s own immune system can turn
against itself to produce auto-antibodies against AQP4, a protein
on certain cells in the optic nerve, brain and spinal cord that are
critical for the survival of nerve cells. The binding of these
anti-AQP4 auto-antibodies activates the complement cascade, another
part of the immune system. Complement activation by anti-AQP4
auto-antibodies can cause destruction of vital cells in the CNS,
leading to demyelination and to the death of neurons, predominantly
in the spinal cord and optic nerve.
About SOLIRIS® SOLIRIS® (eculizumab) is a first-in-class
complement inhibitor that works by inhibiting the C5 protein in the
terminal part of the complement cascade, a part of the immune
system. The terminal complement cascade, when activated in an
uncontrolled manner, plays a role in severe rare and ultra-rare
disorders. SOLIRIS, an intravenously administered therapy, is
approved in the U.S., EU, Japan and other countries as a treatment
for adult patients with PNH and for adults and children with aHUS.
SOLIRIS is not indicated for the treatment of patients with
Shiga-toxin E. coli-related hemolytic uremic syndrome (STEC-HUS).
In the U.S., SOLIRIS is also approved for the treatment of
generalized MG (gMG) in adult patients who are anti-AChR
antibody-positive and for the treatment of neuromyelitis optica
spectrum disorder (NMOSD) in adult patients who are anti-AQP4
antibody-positive. In the EU, SOLIRIS is approved as the first and
only treatment of refractory gMG in adults who are anti-AChR
antibody-positive and for the treatment of NMOSD in adult patients
who are anti-aquaporin-4 (AQP4) antibody-positive with a relapsing
course of the disease. In Japan SOLIRIS is approved for the
treatment of patients with gMG who are anti-AChR antibody-positive
and whose symptoms are difficult to control with high-dose
intravenous immunoglobulin (IVIG) therapy or plasmapheresis
(PLEX).
Important Safety Information
INDICATIONS & IMPORTANT SAFETY INFORMATION FOR SOLIRIS®
(eculizumab)
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.
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 therapies. 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 second complement
inhibitor, a copper-binding agent for Wilson disease and an
anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G
(IgG)-mediated diseases as well as several early-stage therapies,
including one for light chain (AL) amyloidosis and a second
anti-FcRn therapy. 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.
[ALXN-G]
Forward-Looking Statement This press release contains
forward-looking statements that involve risks and uncertainties
relating to future events and the future performance of Alexion,
including statements related to: the potential benefits of SOLIRIS
as a treatment and prevention of relapse in adult patients with
anti-aquaporin-4 (AQP4) antibody-positive neuromyelitis optica
spectrum disorder (NMOSD) and the potential impact of SOLIRIS on
patients suffering from NMOSD; and that Soliris can help prevent
future relapses. Forward-looking statements are subject to factors
that may cause Alexion's results and plans to differ materially
from those expected by these forward looking statements, including
for example: the anticipated benefits of SOLIRIS for NMOSD patients
may not be realized (and the results of the clinical trials may not
be indicative of the results once approved for use in the European
Union); results of clinical trials may not be sufficient to satisfy
any other regulatory authority in order to approve SOLIRIS as a
treatment for NMOSD (or they may request additional trials or
additional information); results in clinical trials may not be
indicative of results from later stage or larger clinical trials
(or in broader patient populations); the possibility that results
of clinical trials are not predictive of safety and efficacy and
potency of our products (or we fail to adequately operate or manage
our clinical trials) which could cause us to discontinue sales of
the product (or halt trials, delay or prevent us from making
regulatory approval filings or result in denial of approval of our
product candidates); unexpected delays in clinical trials;
unexpected concerns regarding products and product candidates that
may arise from additional data or analysis obtained during clinical
trials or obtained once used by patients following product
approval; future product improvements may not be realized due to
expense or feasibility or other factors; delays (expected or
unexpected) in the time it takes regulatory agencies to review and
make determinations on applications for the marketing approval of
our products; inability to timely submit (or failure to submit)
future applications for regulatory approval for our products and
product candidates; inability to timely initiate (or failure to
initiate) and complete future clinical trials due to safety issues,
IRB decisions, CMC-related issues, expense or unfavorable results
from earlier trials (among other reasons); our dependence on sales
from Soliris, our principle product; future competition from
biosimilars and novel products; decisions of regulatory authorities
regarding the adequacy of our research, marketing approval or
material limitations on the marketing of our products; delays or
the inability to launch product candidates due to regulatory
restrictions, anticipated expense or other matters; interruptions
or failures in the manufacture and supply of our products and our
product candidates; failure to satisfactorily address matters
raised by the European Commission and other regulatory agencies
regarding products and product candidates; uncertainty of long-term
success in developing, licensing or acquiring other product
candidates or additional indications for existing products;
inability to complete acquisitions or grow the product pipeline
through acquisitions (including due to failure to obtain antitrust
approvals); the possibility that current rates of adoption of our
products are not sustained; the adequacy of our pharmacovigilance
and drug safety reporting processes; failure to protect and enforce
our data, intellectual property and proprietary rights and the
risks and uncertainties relating to intellectual property claims,
lawsuits and challenges against us (including intellectual property
lawsuits relating to Ultomiris brought by third parties and inter
partes reviews of existing patents); the risk that third party
payors (including governmental agencies) will not reimburse or
continue to reimburse for the use of our products at acceptable
rates or at all; failure to realize the benefits and potential of
investments, collaborations, licenses and acquisitions; the
possibility that expected tax benefits will not be realized;
potential declines in sovereign credit ratings or sovereign
defaults in countries where we sell our products; delay of
collection or reduction in reimbursement due to adverse economic
conditions or changes in government and private insurer regulations
and approaches to reimbursement; uncertainties surrounding legal
proceedings, company investigations and government investigations,
including investigations of Alexion by the U.S. Securities and
Exchange Commission (SEC) and U.S. Department of Justice; the risk
that estimates regarding the number of patients with PNH, aHUS,
gMG, NMOSD, HPP and LAL-D and other indications we are pursuing are
inaccurate; the risks of changing foreign exchange rates; risks
relating to the potential effects of the Company's restructuring;
risks related to the acquisition of Syntimmune and other companies
and co-development efforts; and a variety of other risks set forth
from time to time in Alexion's filings with the SEC, including but
not limited to the risks discussed in Alexion's Quarterly Report on
Form 10-Q for the quarter ended September 30, 2019 and in our other
filings with the SEC. Alexion disclaims any obligation to update
any of these forward-looking statements to reflect events or
circumstances after the date hereof, except when a duty arises
under law.
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version on businesswire.com: https://www.businesswire.com/news/home/20191122005192/en/
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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