Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN) announced today
that the U.S. Food and Drug Administration (FDA) has accepted for
review the Company’s supplemental Biologics License Application
(sBLA) 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 sBLA submission is supported by
comprehensive data from the Phase 3 REGAIN study. The FDA set a
Prescription Drug User Fee Act (PDUFA) date of October 23,
2017.
“We look forward to working with the FDA to bring this
potentially life-transforming treatment to patients who are in dire
and urgent need of effective treatment,” said Martin Mackay, Ph.D.,
Executive Vice President and Global Head of R&D at Alexion.
“Refractory gMG is an ultra-rare disease. Despite existing
treatment options for gMG, patients with refractory gMG 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. Soliris
has received Orphan Drug Designation (ODD) for the treatment of
patients with MG in the U.S. and EU. 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 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, 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).
More information on Soliris, including the full U.S. prescribing
information, is available at www.soliris.net.
Important Safety Information
The U.S. product label for Soliris includes a boxed warning:
“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 [see Warnings and Precautions (5.1)]. Comply with the
most current Advisory Committee on Immunization Practices (ACIP)
recommendations for meningococcal vaccination in patients with
complement deficiencies. Immunize patients with a meningococcal
vaccine 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. [See Warnings and
Precautions (5.1) for additional guidance on the management of the
risk of 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 [see
Warnings and Precautions (5.2)]. 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.”
In patients with PNH, the most frequently reported adverse
events observed with Soliris treatment in clinical studies were
headache, nasopharyngitis (runny nose), back pain and nausea.
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. 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.
Soliris is not indicated for the treatment of patients with
Shiga-toxin E. coli-related hemolytic uremic syndrome
(STEC-HUS). Please see full prescribing information for Soliris,
including BOXED WARNING regarding risk of serious meningococcal
infection.
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.MediaStephanie Fagan,
475-230-3777Senior Vice President, Corporate CommunicationsorArne
Naeveke, PhD, 475-230-3774Executive Director, Product
CommunicationsorInvestorsElena Ridloff, CFA, 475-230-3601Vice
President, Investor Relations
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