Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN) announced today
that it has submitted an application to Japan’s Ministry of Labour
and Welfare (MHLW) to extend the indication for
Soliris® (eculizumab) as a potential treatment for patients
with refractory generalized myasthenia gravis (gMG) who are
anti-acetylcholine receptor (AChR) antibody-positive. The
submission is supported by comprehensive data from the Phase 3
REGAIN study.
“We look forward to working with the Japanese health authorities
to bring this potentially life-transforming treatment to patients
in Japan who are suffering and in urgent need of an effective
therapy,” said Martin Mackay, Ph.D., Executive Vice President and
Global Head of R&D at Alexion. “If approved, Soliris could be a
critical step toward improving the lives of patients with
refractory gMG.”
Patients with AChR-positive refractory gMG represent an
ultra-rare population. Despite existing treatment options for gMG,
these patients continue to face severe complications, including
difficulty walking, talking, swallowing, and breathing normally.
Exacerbations of their disease may be life-threatening and require
hospitalization and intensive care.
If approved, Soliris would be the first and only complement
inhibitor for patients with refractory AChR-positive gMG in Japan.
Alexion has also submitted marketing applications in the U.S. and
EU to extend the indication for Soliris as a potential treatment
for patients with refractory gMG who are AChR-positive, which have
been accepted by the U.S. Food and Drug Administration (FDA) and
validated by the European Medicines Agency (EMA). Soliris has
received Orphan Drug Designation (ODD) for the treatment of
patients with MG in the U.S. and EU, and for treatment of patients
with refractory gMG in Japan. Soliris is not approved in any
country for the treatment of patients with refractory AChR-positive
gMG.
About Refractory Generalized Myasthenia Gravis1-8
Refractory generalized myasthenia gravis (gMG) patients who are
anti-acetylcholine receptor (AChR) antibody-positive represent an
ultra-rare segment of patients with MG—a chronic, debilitating and
progressive autoimmune neuromuscular disease where the complement
system mediates a progressive, destructive inflammatory effect on
the neuromuscular junction. Patients with refractory AChR-positive
gMG experience severe morbidities and life-threatening
complications despite currently available MG therapies.
MG typically begins with weakness in the ocular muscles and
often progresses to the more severe and generalized form, known as
gMG, to include weakness of the head, neck, trunk, limb and
respiratory muscles. While most symptoms in patients with gMG are
managed with conventional therapies, 10% to 15% of patients are
considered refractory—meaning they do not respond to multiple
conventional therapies and continue to suffer profound muscle
weakness throughout the body that can result in slurred speech,
impaired swallowing and choking, double vision, disabling fatigue,
shortness of breath due to respiratory muscle weakness, immobility
requiring assistance, frequent hospital and intensive care unit
admissions with prolonged stays, and periods of respiratory
failure.
In patients with AChR-positive MG, the body’s own immune system
turns on itself to produce antibodies against AChR, a receptor
located on muscle cells in the neuromuscular junction (NMJ) and
used by nerve cells to communicate with the muscles these nerves
control. The binding of these antibodies to AChR activates the
complement cascade which leads to the destruction of the NMJ. As a
result, the communication between nerve and muscle is disrupted,
which leads to a loss of normal muscle function.
Today, there are no approved therapies for the ultra-rare
population of patients suffering from refractory AChR-positive
gMG.
About Soliris® (eculizumab)
Soliris is a first-in-class terminal complement inhibitor
developed from the laboratory through regulatory approval and
commercialization by Alexion. Soliris is approved in the U.S.
(2007), European Union (2007), Japan (2010) and other countries as
the first and only treatment for patients with paroxysmal nocturnal
hemoglobinuria (PNH) to reduce hemolysis. PNH is a debilitating,
ultra-rare and life-threatening blood disorder, characterized by
complement-mediated hemolysis (destruction of red blood cells).
Soliris is also approved in the U.S. (2011), European Union (2011),
Japan (2013) and other countries as the first and only treatment
for patients with atypical hemolytic uremic syndrome (aHUS) to
inhibit complement-mediated thrombotic microangiopathy, or TMA
(blood clots in small vessels). aHUS is a debilitating, ultra-rare
and life-threatening genetic disorder characterized by
complement-mediated TMA. Soliris is not indicated for the treatment
of patients with Shiga-toxin E. coli-related hemolytic uremic
syndrome (STEC-HUS). For the breakthrough medical innovation in
complement inhibition, Alexion and Soliris have received some of
the pharmaceutical industry's highest honors: the Prix Galien USA
(2008, Best Biotechnology Product) and France (2009, Rare Disease
Treatment).
For more information on Soliris, please see full prescribing
information for Soliris, including BOXED WARNING regarding risk of
serious meningococcal infection, available at www.soliris.net.
Important Safety Information
The U.S. prescribing information for Soliris includes the
following warnings and precautions: Life-threatening and fatal
meningococcal infections have occurred in patients treated with
Soliris. Meningococcal infection may become rapidly
life-threatening or fatal if not recognized and treated early.
Comply with the most current Centers for Disease Control (CDC)’s
Advisory Committee on Immunization Practices (ACIP) recommendations
for meningococcal vaccination in patients with complement
deficiencies. Immunize patients with meningococcal vaccines at
least two weeks prior to administering the first dose of Soliris,
unless the risks of delaying Soliris therapy outweigh the risk of
developing a meningococcal infection. Monitor patients for early
signs of meningococcal infections and evaluate immediately if
infection is suspected. Soliris is available only through a
restricted program under a Risk Evaluation and Mitigation Strategy
(REMS). Under the Soliris REMS, prescribers must enroll in the
program. Enrollment in the Soliris REMS program and additional
information are available by telephone: 1-888-SOLIRIS
(1-888-765-4747) or at www.solirisrems.com.
Patients may have increased susceptibility to infections,
especially with encapsulated bacteria. Aspergillus infections have
occurred in immunocompromised and neutropenic patients. Children
treated with Soliris may be at increased risk of developing serious
infections due to Streptococcus pneumoniae and Haemophilus
influenza type b (Hib). Soliris treatment of patients with PNH
should not alter anticoagulant management because the effect of
withdrawal of anticoagulant therapy during Soliris treatment has
not been established. Administration of Soliris may result in
infusion reactions, including anaphylaxis or other hypersensitivity
reactions.
In patients with PNH, the most frequently reported adverse
events observed with Soliris treatment in clinical studies were
headache, nasopharyngitis, back pain and nausea. In patients with
aHUS, the most frequently reported adverse events observed with
Soliris treatment in clinical studies were headache, diarrhea,
hypertension, upper respiratory infection, abdominal pain,
vomiting, nasopharyngitis, anemia, cough, peripheral edema, nausea,
urinary tract infections, and pyrexia.
About Alexion
Alexion is a global biopharmaceutical company focused on
developing and delivering life-transforming therapies for patients
with devastating and rare disorders. Alexion is the global leader
in complement inhibition and has developed and commercializes the
first and only approved complement inhibitor to treat patients with
paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic
uremic syndrome (aHUS), two life-threatening ultra-rare disorders.
In addition, Alexion’s metabolic franchise includes two highly
innovative enzyme replacement therapies for patients with
life-threatening and ultra-rare disorders, hypophosphatasia (HPP)
and lysosomal acid lipase deficiency (LAL-D). Alexion is advancing
its rare disease pipeline with highly innovative product candidates
in multiple therapeutic areas. This press release and further
information about Alexion can be found at: www.alexion.com.
[ALXN-G]
Forward-Looking Statement
This news release contains forward-looking statements, including
statements related to the potential medical benefits of Soliris®
(eculizumab) for the treatment of myasthenia gravis, and Alexion's
future clinical, regulatory and commercial plans for Soliris for
the treatment of myasthenia gravis. Forward-looking statements are
subject to factors that may cause Alexion's results and plans to
differ from those expected, including for example, decisions of
regulatory authorities regarding the adequacy of our research,
marketing approval or material limitations on the marketing of our
products, delays, interruptions or failures in the manufacture and
supply of our products and our product candidates, failure to
satisfactorily address matters raised by the FDA and other
regulatory agencies, the possibility that results of clinical
trials are not predictive of safety and efficacy results of our
products in broader patient populations, the possibility that
current rates of adoption of Soliris in PNH, aHUS or other diseases
are not sustained, the possibility that clinical trials of our
product candidates could be delayed, the adequacy of our
pharmacovigilance and drug safety reporting processes, 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, risks regarding government
investigations, including investigations of Alexion by the SEC and
DOJ, the risk that anticipated regulatory filings are delayed, the
risk that estimates regarding the number of patients with PNH,
aHUS, HPP and LAL-D are inaccurate, the risks of shifting foreign
exchange rates, and a variety of other risks set forth from time to
time in Alexion's filings with the U.S. Securities and Exchange
Commission, including but not limited to the risks discussed in
Alexion's Annual Report on Form 10-K for the period ended December
31, 2016 and in our other filings with the U.S. Securities and
Exchange Commission. Alexion does not intend to update any of these
forward-looking statements to reflect events or circumstances after
the date hereof, except when a duty arises under law.
References
1. Suh J, Goldstein JM, Nowak RJ. Clinical
characteristics of refractory myasthenia gravis patients. Yale J
Biol Med. 2013; 86:255-60. 2. Gilhus NE, Verschuuren JJ. Myasthenia
gravis: subgroup classification and therapeutic strategies. Lancet
Neurol. 2015;14:1023-36. 3. Drachman D, Adams R, Hu R, Jones R,
Brodsky R. Rebooting the immune system with high-dose
cyclophosphamide for treatment of refractory myasthenia gravis. Ann
NY Acad Sci. 2008;1132:305-314. 4. Sathasivam S. Diagnosis and
management of myasthenia gravis. Prog Neurol Psychiatry.
2014;18(1):6-14. 5. Howard JF, ed. Myasthenia Gravis: A Manual for
the Health Care Provider. St. Paul, MN: Myasthenia Gravis
Foundation of America, Inc.; 2008. 6. Safety and efficacy of
eculizumab in refractory generalized myasthenia gravis (REGAIN
study). Clinicaltrials.gov identifier NCT01997229. 7. Melzer N,
Ruck T, Fuhr P, et al. Clinical features, pathogenesis, and
treatment of myasthenia gravis: a supplement to the Guidelines of
the German Neurological Society. J Neurol. 2016 Feb 17. [Epub ahead
of print] 8. Targeting the Complement System in Refractory
Myasthenia Gravis. Supplement to Neurology Reviews. February 2016.
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Alexion Pharmaceuticals, Inc.MediaArne Naeveke, PhD,
475-230-3774Executive Director, Product
CommunicationsorInvestorsElena Ridloff, CFA, 475-230-3601Vice
President, Investor Relations
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