First and Only Complement-based Therapy
Approved for an Ultra-rare Subset of gMG
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) announced today that
the European Commission (EC) approved the extension of the
indication for Soliris® (eculizumab) to include the treatment of
refractory generalized myasthenia gravis (gMG) in adults who are
anti-acetylcholine receptor (AChR) antibody-positive. Soliris is
the first and only complement-based therapy approved in the
European Union (EU) for this ultra-rare subset of patients.1-4
Patients with refractory gMG can have difficulties walking,
talking, swallowing and breathing normally despite therapies
currently used for MG. Exacerbations and crises of their disease
may require hospitalization and intensive care and may be
life-threatening.5-7 Soliris will be launched for this new
indication initially in Germany, and Alexion is evaluating launches
in additional EU countries.
“Patients with refractory gMG have exhausted multiple therapies
and continue to suffer from severe symptoms and complications that
markedly impact their daily lives,” said Renato Mantegazza, MD,
from the Department of Neuroimmunology and Neuromuscular Diseases,
at the Istituto Neurologico Carlo Besta in Milan, Italy, and an
investigator in the Phase 3 REGAIN study. “There is an urgent need
for therapy for these patients, and it’s exciting to have a product
such as Soliris available that has demonstrated in clinical studies
that it improves patients’ symptoms and their ability to undertake
daily activities.”
Chronic uncontrolled activation of the complement cascade, a
part of the immune system, can play a major role in the
debilitating symptoms and potentially life-threatening
complications of refractory gMG.8-10 Soliris is a first-in-class
complement inhibitor that specifically and effectively inhibits the
terminal part of the complement cascade.
“Our deep understanding of complement-mediated diseases enabled
us to develop Soliris for the treatment of patients with refractory
gMG,” said John Orloff, M.D., Executive Vice President and Head of
Research & Development at Alexion. “We are grateful to the
investigators and patients who participated in our clinical
program, and we are excited about the opportunity to bring Soliris
to patients who continue to suffer from this debilitating disease
despite current therapies.”
The EC based its approval of the extended indication for Soliris
on comprehensive clinical data from the Phase 3 REGAIN study
(MG-301) and its long-term open-label extension study (MG-302).
Alexion’s supplemental Biologics License Application (sBLA) in
the U.S. and a supplemental new drug application in Japan for
Soliris as a treatment for patients with anti-AChR
antibody-positive refractory gMG have been accepted for review by
the U.S. Food and Drug Administration (FDA) and the Japanese
Ministry of Health, Labour and Welfare (MHLW), respectively.
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 Refractory Generalized Myasthenia Gravis
Patients with refractory generalized myasthenia gravis (gMG) who
are anti-acetylcholine receptor (AChR) antibody-positive represent
an ultra-rare subset of MG patients1-4 who continue to suffer from
severe disease symptoms and complications despite therapies
currently used for MG.1-2,11
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
60.5,6,12,13 It typically begins with weakness in the muscles that
control the movements of the eyeballs 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.13
While most symptoms in patients with gMG are managed with
therapies for MG, 10% to 15% of patients are considered
refractory—meaning they do not respond to multiple therapies for MG
and continue to suffer profound muscle weakness, and severe disease
symptoms that limit function.1-2,11 Patients with refractory gMG
can suffer from slurred speech; 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 life-threatening.5-7
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 in the neuromuscular junction
(NMJ) and used by nerve cells to communicate with the muscles these
nerves control.5,6 The binding of these antibodies to AChR
activates the complement cascade, another part of the immune
system, which leads to a localized destruction of the NMJ. As a
result, the communication between nerve and muscle is impaired,
which in turn leads to a loss of normal muscle function.8-10,14
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
refractory generalized myasthenia gravis (gMG).
Soliris is approved in the U.S., EU, Japan and other countries
as the first and only treatment for patients with PNH and aHUS, and
in the EU as the first and only treatment for refractory gMG in
patients 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
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), atypical hemolytic
uremic syndrome (aHUS), and refractory 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.
[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 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, 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 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.
References
- Silvestri N, Wolfe G.
Treatment-refractory myasthenia gravis. J. Clin Neuromuscul Dis.
2014;15(4):167-178.
- Howard J. Targeting the Complement
System in Refractory Myasthenia Gravis. Supplement to Neurology
Reviews. February 2016.
- Suh J., Goldstein JM, Nowak RJ.
Clinical Characteristics of Refractory Myasthenia Gravis Patients.
Yale J Biol Med. 2013;86(2):255-260.
- Regulation (EU) No 536/2014 of the
European Parliament and of the Council of 16 April 2014 on clinical
trials on medicinal products for human use, and repealing Directive
2001/20/EC.
http://eur-lex.europa.eu/legal-content/EN/TXT/PDF/?uri=CELEX:32000R0141&qid=1421232987002&from=EN.
Accessed on June 26, 2017.
- 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. 2013;48(1):76-84.
- 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.
- Sathasivam S. Diagnosis and management
of myasthenia gravis. Progress in Neurology and Psychiatry.
January/February 2014.
- Tüzün E, Huda R, Christadoss P.
Complement and cytokine based therapeutic strategies in myasthenia
gravis. JAutoimmun. 2011;37(2):136-143.
- Meriggioli MN, Sanders DB. Muscle
autoantibodies in myasthenia gravis: beyond diagnosis? Expert Rev.
Clin.Immunol. 2012;8(5), 427-428.
- Conti-Fine, et al. Myasthenia gravis:
past, present, and future. J Clin Invest. 2006; 116:2843-2354.
- Sanders DB, Wolfe, GI, Benatar M, et
al. International consensus guidance for management of myasthenia
gravis: Executive summary. Neurology. 2016 Jul
26;87(4):419-25.
- Huda R, Tüzün E, Christadoss P.
Targeting complement system to treat myasthenia gravis. Rev.
Neurosci. 2014; 25(4): 575–583.
- Meriggioli MN, Sanders DB. Autoimmune
myasthenia gravis: emerging clinical and biological heterogeneity.
Lancet Neurol. 2009-8(5): 475-490.
- Buzzard, K. A., N. J. Meyer, T. A.
Hardy, D. S. Riminton and S. W. Reddel. Induction intravenous
cyclophosphamide followed by maintenance oral immunosuppression in
refractory myasthenia gravis. Muscle Nerve. 2015;52(2):
204-210.
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Alexion Pharmaceuticals, Inc.MediaArne Naeveke, PhD,
475-230-3774Executive Director, Product
CommunicationsorInvestorsElena Ridloff, CFA, 475-230-3601Vice
President, Investor RelationsorCatherine Hu, 475-230-3599Director,
Investor Relations
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