Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that
Soliris® (eculizumab) has been granted orphan drug designation
(ODD) by Japan’s Ministry of Health, Labour and Welfare (MHLW) for
the treatment of patients with myasthenia gravis (MG), a rare,
debilitating neurologic disorder. In patients with MG, uncontrolled
complement activation due to antibodies directed at the
neuromuscular junction can ultimately lead to profound and
debilitating weakness of various muscle groups throughout the
body.1,2 This significant muscle weakness can impair patients’
ability to walk, speak clearly, swallow and, in some cases, to
breathe.
“The orphan drug designation for eculizumab for MG highlights
the significant need for an effective treatment option for patients
in Japan who continue to suffer from the severe and debilitating
symptoms of MG despite currently available therapies,” said Martin
Mackay, Ph.D., executive vice president and global head of R&D
at Alexion. “By specifically inhibiting the terminal complement
pathway, eculizumab has the potential to provide better treatment
outcomes for patients with refractory generalized MG. We look
forward to evaluating the clinical benefits of eculizumab in MG in
our registration study, known as REGAIN, which is currently
enrolling patients.”
The MHLW, based on the opinion of the Pharmaceutical Affairs and
Food Sanitation Council, grants orphan status to drugs and medical
devices that treat serious diseases of high unmet medical need that
affect fewer than 50,000 patients in Japan. ODD provides drug
developers with certain benefits and incentives, including priority
review for marketing authorization and a period of 10 years of
market exclusivity if regulatory approval is received for the
designated indication.
Alexion is enrolling patients in a multinational,
placebo-controlled registration trial of eculizumab in patients
with refractory generalized MG, known as the REGAIN (Eculizumab for
REfractory GenerAlIzed
MyastheNia Gravis) study. More
information on this trial is available
at www.clinicaltrials.gov under the identifier
NCT01997229.
Soliris is a first-in-class terminal complement inhibitor and is
currently approved in the United States, European Union, Japan and
other countries for the treatment of patients with paroxysmal
nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic
syndrome (aHUS), two debilitating, ultra-rare and life-threatening
disorders caused by chronic uncontrolled complement activation.
Soliris is not approved in any country for the treatment of MG. In
2014, Soliris was granted ODD in both the U.S. and EU for the
treatment of MG.
About Myasthenia Gravis
Myasthenia gravis (MG) is a rare, debilitating neurologic
disorder caused by auto-antibodies that recognize a specific target
in the nerve-muscle junction, which results in life-long
uncontrolled terminal complement activation causing tissue damage
and interference with signaling between nerve and muscle
fibers.1,2 Patients with MG initially experience weakness in
their ocular (eye) muscles, and the disease typically progresses to
the more severe and generalized form to include weakness of head,
trunk, limb and respiratory muscles. Symptoms can include drooping
eyelid, weakness in the arms and legs, slurred speech, difficulty
chewing or swallowing, and difficulty breathing, which could lead
to a life-threatening myasthenic crisis.
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), the 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 including the full U.S. prescribing information
on Soliris 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)."
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, 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 biopharmaceutical company focused on serving
patients with severe and rare disorders through the innovation,
development and commercialization of life-transforming therapeutic
products. Alexion is the global leader in complement inhibition and
has developed and markets Soliris® (eculizumab) as a treatment for
patients with PNH and aHUS, two debilitating, ultra-rare and
life-threatening disorders caused by chronic uncontrolled
complement activation. Soliris is currently approved in nearly 50
countries for the treatment of PNH, and in nearly 40 countries for
the treatment of aHUS. Alexion is evaluating other potential
indications for Soliris in additional severe and ultra-rare
disorders beyond PNH and aHUS, and is developing other highly
innovative biotechnology product candidates across multiple
therapeutic areas. This press release and further information about
Alexion can be found at www.alexionpharma.com.
[ALXN-G]
This news release contains forward-looking statements, including
statements related to potential medical benefits of Soliris®
(eculizumab) for the treatment of myasthenia gravis (MG).
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 marketing approval or material limitations on the
marketing of Soliris for MG, delays in arranging satisfactory
manufacturing capabilities, the possibility that results of
clinical trials are not predictive of safety and efficacy results
of Soliris for MG in broader or different patient populations,
decisions of regulatory authorities to require additional testing,
the risk that estimates regarding the number of patients with MG
and observations regarding the natural history of patients with MG
are inaccurate, and a variety of other risks set forth from time to
time in Alexion's filings with the 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
September 30, 2014. 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. Conti-Fine BM, Milani M, Kaminski HJ. Myasthenia gravis:
past, present, and future. J Clin Invest 2006;116(11):2843-54.
2. Tüzün E, Huda R, Christadoss P. Complement and cytokine based
therapeutic strategies in myasthenia gravis. J Autoimmun
2011;37(2):136-43.
Alexion:MediaIrving Adler, 203-271-8210Executive Director,
Corporate CommunicationsorKim Diamond, 203-439-9600Senior Director,
Corporate CommunicationsorInvestorsElena Ridloff, CFA,
203-699-7722Executive Director, Investor Relations
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