NEW HAVEN, Conn.--(BUSINESS WIRE)--
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) announced today that the
Committee for Medicinal Products for Human Use (CHMP) of the European
Medicines Agency (EMA) has adopted a positive opinion to extend the
current therapeutic indication for Soliris® (eculizumab) to
include the treatment of refractory generalized myasthenia gravis (gMG)
in patients who are anti-acetylcholine receptor (AChR)
antibody-positive. The final decision from the European Commission (EC)
is anticipated in the third quarter of 2017. If approved, Soliris will
be the first treatment available in the European Union (EU) for patients
with refractory gMG who are anti-AChR antibody-positive, and the first
and only complement inhibitor approved for this disease.
"Despite existing treatment options for gMG, patients with refractory
gMG continue to suffer from severe symptoms and disease complications
that significantly impact their daily lives," said John Orloff, M.D.,
Executive Vice President and Head of Research & Development at Alexion.
"The positive CHMP opinion is a critical milestone in bringing Soliris
to patients with refractory gMG who are anti-AChR antibody-positive and
for whom physicians currently have no approved therapy."
Patients with refractory gMG have difficulties walking, talking,
swallowing and breathing normally. Exacerbations and crises of their
disease may require hospitalization and intensive care and may be
life-threatening. Patients with refractory gMG who are anti-AChR
antibody-positive represent an ultra-rare segment of patients with
myasthenia gravis (MG).1-10
The CHMP based its opinion on comprehensive clinical data from the Phase
3 REGAIN study (MG-301) and its long-term extension study (MG-302). A
summary of the CHMP opinion can be accessed here.
Soliris is approved in the United States (U.S.), EU, Japan and other
countries for the treatment of patients with paroxysmal nocturnal
hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), two
ultra-rare, complement-mediated disorders. 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
About Refractory Generalized Myasthenia Gravis
Patients with refractory generalized myasthenia gravis (gMG) who are
anti-acetylcholine receptor (AChR) antibody-positive represent an
ultra-rare segment of patients with myasthenia gravis (MG) who continue
to suffer from severe disease symptoms and complications despite
available treatment options for gMG. There are no approved therapies for
patients with anti-AChR antibody-positive refractory gMG.1-3
MG is a chronic, debilitating and progressive autoimmune neuromuscular
disease that typically begins with weakness in the ocular muscles and
often progresses to the more severe and generalized form, known as gMG,
which includes weakness of the head, neck, trunk, limb and respiratory
muscles.4-6 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, double or blurred vision, disabling fatigue, shortness of
breath, immobility requiring assistance, frequent hospital and intensive
care unit admissions with prolonged stays and periods of respiratory
failure. Complications, exacerbations and crises of refractory gMG can
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. The
binding of these antibodies to AChR activates the complement cascade,
another part of the immune system, which leads to progressive
inflammatory damage at the NMJ. As a result, the communication between
nerve and muscle is impaired, which in turn leads to a loss of normal
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 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
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.
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, 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 generalized Myasthenia
Gravis (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,
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, risks related to potential disruptions to our
business as a result of leadership changes, 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 March 31, 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.
1. Carr A, Cardwell C, McCarron P, et al. A systemic review of
population based epidemiological studies in Myasthenia Gravis. BMC
Neurology. 2010, 10:46.
2. Silvestri N, Wolfe G. Treatment-refractory myathenia gravis. J. Clin
Neuromuscul Dis. 2014;15(4):167-178.
3. 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.
4. Huda R, Tüzün E, Christadoss P. Targeting complement system to treat
myasthenia gravis Rev. Neurosci. 2014; 25(4): 575-583
5. 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.
6. Meriggioli MN, Sanders DB. Autoimmune myasthenia gravis: emerging
clinical and biological heterogeneity. Lancet Neurol. 2009-8(5): 475-490.
7. Howard J. Targeting the Complement System in Refractory Myasthenia
Gravis. Supplement to Neurology Reviews. February 2016.
8. National Institute of Neurological Disorders and Stroke. Myasthenia
Gravis Fact Sheet. Last modified May 10, 2016. http://www.ninds.nih.gov/disorders/myasthenia_gravis/detail_myasthenia_gravis.htm.
Accessed May 31, 2016.
9. Sathasivam S. Diagnosis and management of myasthenia gravis. Progress
in Neurology and Psychiatry. January/February 2014.
10. Howard JF. Myasthenia gravis: A manual for the healthcare provider.
Myasthenia Gravis Foundation of America, Inc. 2008.
11. Conti-Fine BM, Milani M, Kaminski HJ. Myasthenia gravis: past,
present, and future. J Clin Invest. 2006;116(11):2843-2854.
12. Tüzün E, Huda R, Christadoss P. Complement and cytokine based
therapeutic strategies in myasthenia gravis. J Autoimmun.
13. Meriggioli MN, Sanders DB. Muscle autoantibodies in myasthenia
gravis: beyond diagnosis? Expert Rev. Clin. Immunol. 2012-8(5), 427-428
View source version on businesswire.com: http://www.businesswire.com/news/home/20170623005262/en/
Alexion Pharmaceuticals, Inc.
Arne Naeveke, PhD,
Executive Director, Product Communications
Ridloff, CFA, 475-230-3601
Vice President, Investor Relations
Director, Investor Relations
Source: Alexion Pharmaceuticals, Inc.
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