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 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 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 be
life-threatening.5,7-10
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 muscle function.4,5,8,11-13
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
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, 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.
References
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.
2011;37(2):136-143.
13. Meriggioli MN, Sanders DB. Muscle autoantibodies in
myasthenia gravis: beyond diagnosis? Expert Rev. Clin. Immunol.
2012-8(5), 427-428
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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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