First FDA-Approved Treatment in More Than 60 Years for Patients with
gMG, a Chronic and Debilitating Neuromuscular Disorder
NEW HAVEN, Conn.--(BUSINESS WIRE)--
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) announced today that the
U.S. Food and Drug Administration (FDA) has approved Soliris®
(eculizumab) as a treatment for adult patients with generalized
myasthenia gravis (gMG) who are anti-acetylcholine receptor (AchR)
antibody-positive. In the Phase 3 REGAIN study and its ongoing
open-label extension study, Soliris demonstrated treatment benefits for
patients with anti-AchR antibody-positive gMG who had previously failed
immunosuppressive treatment and continued to suffer from significant
unresolved disease symptoms, which can include difficulties seeing,
walking, talking, swallowing and breathing. These patients are at an
increased risk of disease exacerbations and crises that may require
hospitalization and intensive care and may be life-threatening. These
patients represent approximately 5-10% of all patients with MG.1-6
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"Today's approval is a significant milestone for Alexion and, more
importantly, for the subset of patients with anti-AchR antibody-positive
gMG who continue to suffer from significant unresolved disease symptoms
despite existing treatment options," said John Orloff, M.D., Executive
Vice President and Head of Research & Development at Alexion. "We are
proud that we could apply our deep understanding of complement biology
to develop Soliris for the treatment of patients with this debilitating
Chronic uncontrolled activation of the complement system, a part of the
immune system, plays a major role in the debilitating symptoms and
potentially life-threatening complications for patients with gMG who are
anti-AchR antibody-positive.1-4 By selectively and
effectively inhibiting the terminal complement cascade, Soliris targets
a critical underlying cause of the disease.
"I am pleased that the FDA recognized the comprehensive clinical data
supporting the benefits of Soliris for patients with anti-AchR
antibody-positive gMG," said Professor James F. Howard, M.D., Department
of Neurology at the University of North Carolina, Chapel Hill, and lead
investigator in the clinical development of this new indication. "It is
exciting that patients who have not responded adequately to existing
therapies will now have a new treatment option that was shown in
clinical studies to improve patients' symptoms, their ability to carry
out activities of daily living and their quality of life."
"This is a landmark day for the members of the U.S. myasthenia gravis
community, who have not seen a therapy approved for generalized
myasthenia gravis in more than 60 years," said Nancy Law, Chief
Executive Officer of the Myasthenia Gravis Foundation of America (MGFA).
"It is particularly significant that this approval of Soliris will
provide a new option for those with gMG and especially for those who do
not respond adequately to or cannot tolerate standard treatment options."
The FDA based its approval of the extended indication for Soliris on
comprehensive clinical data from the Phase 3, randomized, double-blind,
placebo-controlled, multicenter REGAIN study (ECU-MG-301).
Soliris is approved in the European Union (EU) for the treatment of
refractory gMG in adults who are anti-AchR antibody-positive. Alexion's
new drug application in Japan for Soliris as a treatment for patients
with anti-AchR antibody-positive refractory gMG has been accepted for
review by the Japanese Ministry of Health, Labour and Welfare (MHLW).
Soliris has received Orphan Drug Designation (ODD) for the treatment of
patients with MG in the U.S. and EU, and for the treatment of patients
with refractory gMG in Japan.
About Generalized Myasthenia Gravis
Myasthenia gravis (MG) is a debilitating, chronic and progressive
autoimmune neuromuscular disease that can occur at any age but most
commonly begins for women before the age of 40 and men after the age of
60.7-10 It 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 gMG, with weakness of the
head, neck, trunk, limb and respiratory muscles.10
While most patients with gMG can be managed with current therapies for
MG, 10-15% of patients fail to respond adequately to or cannot tolerate
multiple therapies for MG and continue to suffer profound muscle
weakness, and severe disease symptoms that limit function.1,11,12 These
patients can suffer from slurred speech, choking, impaired swallowing,
double or blurred vision, disabling fatigue, immobility requiring
assistance, shortness of breath, and episodes of respiratory failure.
Complications, exacerbations and myasthenic crises can require hospital
and intensive care unit admissions with prolonged stays and can be
In patients with anti-AchR antibody-positive MG, the body's own immune
system turns on itself to produce antibodies against AchR, a receptor
located on muscle cells at the neuromuscular junction (NMJ) and used by
nerve cells to communicate with the muscles these nerves control.8,9
The binding of these antibodies to AchR activates the complement
cascade, another part of the immune system, which leads to a localized
inflammation and destruction of the muscle membrane at the NMJ.14-16
As a result, the communication between nerve and muscle is impaired,
which in turn leads to a loss of normal muscle function.8,9
Patients with anti-AchR antibody-positive gMG who continue to suffer
from severe disease symptoms and complications despite current therapies
for MG represent approximately 5-10% of all patients with MG.7-10
About Soliris® (eculizumab)
Soliris® is a first-in-class complement inhibitor that works
by inhibiting the terminal part of the complement cascade, a part of the
immune system that, when activated in an uncontrolled manner, plays a
role in serious ultra-rare disorders like paroxysmal nocturnal
hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS) and
anti-acetylcholine receptor (AchR) antibody-positive myasthenia gravis
Soliris is approved in the U.S., EU, Japan and other countries as the
first and only treatment for patients with PNH and aHUS, in the EU as
the first and only treatment of refractory gMG in adults who are
anti-AchR antibody-positive, and in the U.S. for the treatment of adult
patients with gMG who are anti-AchR antibody-positive. Soliris is not
indicated for the treatment of patients with Shiga-toxin E. coli-related
hemolytic uremic syndrome (STEC-HUS). Alexion and Soliris have received
some of the pharmaceutical industry's highest honors for the medical
innovation in complement inhibition: 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 Soliris 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
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. In patients with gMG who are anti-AchR antibody-positive, the
most frequently reported adverse reaction observed with Soliris
treatment in the placebo-controlled clinical study (≥10%) was
Alexion is a global biopharmaceutical company focused on serving
patients and families affected by rare diseases through the innovation,
development and commercialization of life-changing therapies. 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), atypical
hemolytic uremic syndrome (aHUS), and anti-acetylcholine receptor (AchR)
antibody-positive generalized myasthenia gravis (gMG). In addition,
Alexion 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). As
the leader in complement biology for over 20 years, 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, and metabolic disorders. This press
release and further information about Alexion can be found at: www.alexion.com.
This news release contains forward-looking statements, including
statements related to the potential medical benefits of Soliris®
(eculizumab) for the treatment of generalized myasthenia gravis (gMG),
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, the risks
and uncertainties of drug development, decisions of regulatory
authorities regarding the adequacy of our research, marketing approval
or material limitations on the marketing of eculizumab for the treatment
of gMG, 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 clinical trials of our product candidates could be
delayed, the adequacy of our pharmacovigilance and drug safety reporting
processes, the risk that third party payers (including governmental
agencies) will not reimburse or continue to reimburse for the use of our
products at acceptable rates or at all, the outcome of challenges and
opposition proceedings to our intellectual property, assertion or
potential assertion by third parties that the manufacture, use or sale
of our products infringes their intellectual property, uncertainties
surrounding legal proceedings, company investigations and 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 gMG are inaccurate, 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 Quarterly Report on Form
10-Q for the period ended June 30, 2017 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.
Silvestri N, Wolfe G. Treatment-refractory myasthenia gravis. J.
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Suh J., Goldstein JM, Nowak RJ. Clinical Characteristics of
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Howard J. Myasthenia Gravis - A summary. Myasthenia Gravis
Foundation of America. http://www.myasthenia.org/HealthProfessionals/ClinicalOverviewofMG.aspx.
Accessed October 12, 2017.
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Souayah N, Nasar A, Suri MF, et al. Int J Biomed Sci.
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Howard JF, Barohn RJ, Cutter GR et al. A randomized, double-blind,
placebo-controlled phase II study of eculizumab in patients with
refractory generalized myasthenia gravis. Muscle Nerve.
National Institute of Neurological Disorders and Stroke.
Myasthenia Gravis Fact Sheet. Publication date May 2017. http://www.ninds.nih.gov/disorders/myasthenia_gravis/detail_myasthenia_gravis.htm.
Meriggioli MN, Sanders DB. Autoimmune myasthenia gravis: emerging
clinical and biological heterogeneity. Lancet Neurol.
Howard J. Targeting the Complement System in Refractory Myasthenia
Gravis. Supplement to Neurology Reviews. February 2016.
Sanders DB, Wolfe, GI, Benatar M, et al. International consensus
guidance for management of myasthenia gravis: Executive summary.
Sathasivam S. Diagnosis and management of myasthenia gravis.
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Conti-Fine, et al. Myasthenia gravis: past, present, and future. J
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Tüzün E, Huda R, Christadoss P. Complement and cytokine based
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gravis: beyond diagnosis? Expert Rev. Clin. Immunol. 2012;8(5),
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Alexion Pharmaceuticals, Inc.
Naeveke, PhD, 475-230-3774
Executive Director, Product
Kim Diamond, 475-230-3775
Director, Corporate Communications
Ridloff, CFA, 475-230-3601
Vice President, Investor Relations
Director, Investor Relations
Source: Alexion Pharmaceuticals, Inc.
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