Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that
researchers will present data from clinical studies of Soliris®
(eculizumab) in patients with paroxysmal nocturnal hemoglobinuria (PNH)
and atypical hemolytic uremic syndrome (aHUS), two life-threatening and
ultra-rare diseases caused by chronic uncontrolled complement
activation, as well as data from the Global aHUS Registry, at the 56th
Annual Meeting of the American Society of Hematology (ASH). Soliris is
the first and only approved treatment for PNH and aHUS. Abstracts
summarizing these presentations are published on the ASH website and can
be accessed using the links below. The ASH annual meeting will be held
December 6-9, 2014, in San Francisco.
Soliris, a first-in-class terminal complement inhibitor, is approved in
nearly 50 countries as a treatment for patients with PNH, a
debilitating, ultra-rare and life-threatening blood disorder
characterized by complement-mediated hemolysis (destruction of red blood
cells), and in nearly 40 countries as a treatment for patients with
aHUS, a genetic, chronic and ultra-rare disease associated with vital
organ failure and premature death.
Soliris and PNH
The following abstract will be presented in a poster session on
Saturday, December 6, 2014, from 5:30 to 7:30 p.m., Pacific Standard
Time (PST):
Abstract 1595: “The Interim Analysis of the OPTIMA (Observation of
GPI-anchored Protein-Deficient [PNH-type] Cells in Japanese Patients
with Bone Marrow Failure Syndrome and in those Suspected of Having PNH)
Study,” Noji, et al.
Accessible at: https://ash.confex.com/ash/2014/webprogram/Paper74188.html
Soliris and aHUS
The following abstract will be presented in a poster session on Sunday,
December 7, 2014, from 6:00 to 8:00 p.m., Pacific Standard Time (PST):
Abstract 2789: “Eculizumab is an Effective Treatment for Atypical
Hemolytic Uremic Syndrome in Pediatric and Adult Patients with or
without Identified Genetic Complement Mutations or Complement Factor H
Autoantibodies,” Cataland, et al.
Accessible at: https://ash.confex.com/ash/2014/webprogram/Paper73345.html
The following abstract will be presented in a poster session on Monday,
December 8, 2014, from 6:00 to 8:00 p.m., Pacific Standard Time (PST):
Abstract 4204: “Baseline Demographics and Characteristics of 466
Patients with Atypical Hemolytic Uremic Syndrome in the Global aHUS
Registry,” Licht, et al.
Accessible at: https://ash.confex.com/ash/2014/webprogram/Paper69197.html
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), a debilitating,
ultra-rare and life-threatening blood disorder, characterized by
complement-mediated hemolysis (destruction of red blood cells). Soliris
is indicated to reduce hemolysis. 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), a debilitating, ultra-rare and life-threatening genetic
disorder characterized by complement-mediated thrombotic
microangiopathy, or TMA (blood clots in small vessels). Soliris is
indicated to inhibit 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
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 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, including asfotase
alfa, across multiple therapeutic areas. This press release and further
information about Alexion can be found at: www.alexionpharma.com.
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Copyright Business Wire 2014