Alexion Joins EURORDIS, NORD and Patient Organizations Worldwide in Celebrating Rare Disease Day 2012
CHESHIRE, Conn.--(BUSINESS WIRE)--
Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) joins the European
Organization for Rare Diseases (EURORDIS), the National Organization for
Rare Disorders (NORD) and patient groups, families, governments, medical
communities and industry in celebrating Rare Disease Day, a global
effort to raise awareness of rare diseases and their profound impact on
patients' lives.
Many rare and ultra-rare diseases are chronic, progressive and marked by
continuing pain, severe disability and high mortality rates. The
severity of rare diseases is often compounded by a lack of scientific
knowledge, research and medical innovation, leading to delayed
diagnoses, misdiagnoses and sub-optimal treatment.
"Too often, patients and families coping with a rare disease feel
isolated and lack information, support programs, access to medical
experts and effective therapies," said Deborah Sittig, Founder of Soft
Bones, the U.S. Hypophosphatasia Foundation. "As the mother of a child
with a severe, ultra-rare metabolic disorder and the founder of a
patient organization, I have experienced the benefits of joining
together with families, physicians and industry to improve the care of
these often underserved patients."
"Rare Disease Day brings much-needed attention to rare diseases as an
important public health issue with unique challenges," said Yann Le Cam,
Chief Executive Officer, EURORDIS. "This year's Rare Disease Day
celebration calls for solidarity in meeting the challenges faced by many
patients with rare diseases, including difficulty obtaining an accurate
diagnosis, limited scientific information, and limited treatment
options."
Helping More Patients With Ultra-Rare Disorders
The goals of Rare Disease Day align with Alexion's mission to develop
innovative, life-transforming therapies for patients with ultra-rare and
severe disorders for which there are few, if any, treatment options.
Alexion discovered and developed Soliris® (eculizumab), the
first treatment for patients with paroxysmal nocturnal hemoglobinuria
(PNH), an ultra-rare, debilitating, and life-threatening blood disorder,
and the first treatment for atypical hemolytic uremic syndrome (aHUS),
an ultra-rare, debilitating, and life-threatening genetic disorder.
Alexion is committed to serving an expanding number of patients with
PNH, aHUS and other ultra-rare disorders worldwide. The Company is
evaluating the benefits of Soliris in several other ultra-rare and
life-threatening disorders, and is simultaneously developing other
highly innovative drug candidates for the treatment of additional
ultra-rare disorders. These include hypophosphatasia (HPP), a
life-threatening, genetic metabolic disorder that leads to progressive
damage to multiple vital organs, and molybdenum cofactor deficiency
(MoCD) Type A, a devastating disorder that leads to severe brain damage
and rapid death in newborns. There are no approved or effective
therapies for HPP or MoCD Type A.
"We applaud EURORDIS, NORD and other patient advocacy groups for their
continued collaboration to serve patients and families affected by
severe and ultra-rare disorders," said Leonard Bell, M.D., Chief
Executive Officer of Alexion. "The employees of Alexion share this
steadfast commitment to bringing hope to these patients and families, by
developing and delivering innovative and effective new therapies."
To learn more about Alexion's research and development programs, and our
commitment to patients with ultra-rare disorders, please visit our
website at www.alexionpharma.com.
To learn more about Rare Disease Day, visit www.rarediseaseday.us for
U.S. activities and www.rarediseaseday.org for
global activities.
Rare and Ultra-Rare Disorders
In the United States a rare disease is designated as rare if it affects
fewer than approximately 650 patients per million of the population. In
the European Union, a disease is designated as rare if it affects fewer
than 500 patients per million of the population. An even rarer set of
diseases are known as ultra-rare, typically designated when there are
fewer than 20 patients per million of the population. Thus in the United
States, while a very prevalent disease may affect millions of people, a
rare disease will affect no more than 200,000 Americans and an
ultra-rare disease will affect approximately 6,000 at most — and often
far fewer.
About Paroxysmal Nocturnal Hemoglobinuria (PNH)
PNH is an ultra-rare blood disorder in which chronic, uncontrolled
activation of complement, a component of the normal immune system,
results in hemolysis (destruction of the patient's red blood cells). PNH
strikes people of all ages, with an average age of onset in the early
30s.1 Approximately 10 percent of all patients first develop
symptoms at 21 years of age or younger.2 PNH develops without
warning and can occur in men and women of all races, backgrounds and
ages. PNH often goes unrecognized, with delays in diagnosis ranging from
one to more than 10 years.3 In the period of time before
Soliris was available, it had been estimated that approximately
one-third of patients with PNH did not survive more than five years from
the time of diagnosis.3 PNH has been identified more commonly
among patients with disorders of the bone marrow, including aplastic
anemia (AA) and myelodysplastic syndromes (MDS).4,5,6 In
patients with thrombosis of unknown origin, PNH may be an underlying
cause.1 More information on PNH is available at www.pnhsource.com.
About Atypical Hemolytic Uremic Syndrome (aHUS)
aHUS is a chronic, ultra-rare, and life-threatening disease in which a
genetic deficiency in one or more complement regulatory genes causes
life-long uncontrolled complement activation, resulting in
complement-mediated thrombotic microangiopathy (TMA), the formation of
blood clots in small blood vessels throughout the body.7,8 Permanent,
uncontrolled complement activation in aHUS causes a life-long risk for
TMA, which leads to sudden, catastrophic, and life-threatening damage to
the kidney, brain, heart, and other vital organs, and premature death. 8,9 More
than half of all patients with aHUS die, require kidney dialysis or have
permanent kidney damage within 1 year of diagnosis.10 The
majority of patients with aHUS who receive a kidney transplant commonly
experience subsequent systemic TMA, resulting in a 90% transplant
failure rate.11
aHUS affects both children and adults. In a large group of aHUS
patients, 60% were first diagnosed at younger than 18 years of age.11 Complement-mediated
TMA also causes reduction in platelet count (thrombocytopenia) and red
blood cell destruction (hemolysis). While mutations have been identified
in at least ten different complement regulatory genes, mutations are not
identified in 30-50% of patients with a confirmed diagnosis of aHUS.12
About Hypophosphatasia (HPP)
HPP is an ultra-rare, genetic, and life-threatening metabolic disease
characterized by defective bone mineralization and impaired phosphate
and calcium regulation leading to progressive damage to multiple vital
organs including destruction and deformity of bones, profound muscle
weakness, seizures, impaired renal function, and respiratory failure.13,14,15,16
The severe manifestations of the genetic deficiency in HPP affect
people of all ages, and approximately 50 percent of infants with the
disease do not survive past one year of age.13
HPP is caused by a genetic deficiency of an enzyme known as tissue
non-specific alkaline phosphatase (TNSALP), which causes life-long
abnormalities in metabolism of the two vital minerals calcium and
phosphate, leading directly to the debilitating morbidities and
premature mortality of the disease.13 There are currently no
therapies approved for HPP.13
About Soliris
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 US, European Union, Japan 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 also approved in
the US and the European Union 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.
The effectiveness of Soliris in aHUS is based on the effects on TMA and
renal function. Prospective clinical trials in additional patients are
ongoing to confirm the benefit of Soliris in patients with aHUS. Soliris
is not indicated for the treatment of patients with Shiga toxin E. coli
related hemolytic uremic syndrome (STEC-HUS). Alexion's breakthrough
approach in complement inhibition has received the pharmaceutical
industry's highest honors: the 2008 Prix Galien USA Award for Best
Biotechnology Product with broad implications for future biomedical
research and the 2009 Prix Galien France Award in the category of Drugs
for Rare Diseases. More information including the full prescribing
information on Soliris is available at www.soliris.net.
Important Safety Information
Soliris is generally well tolerated in patients with PNH and aHUS. 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 hypertension, upper respiratory tract infection, diarrhea,
headache, anemia, vomiting, nausea, urinary tract infection, and
leukopenia.
The U.S. product label for Soliris also 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.
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 2 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 Serious
Meningococcal Infections (5.1) for additional guidance on the management
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 (5.2). Enrollment in the
Soliris REMS program and additional information are available by
telephone: 1-888-soliris (1-888-765-4747)."
Please see full prescribing information for Soliris, including boxed
WARNING regarding risk of serious meningococcal infection.
About Alexion
Alexion Pharmaceuticals, Inc. is a biopharmaceutical company focused on
serving patients with severe and ultra-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 more than 35 countries for
the treatment of PNH, and in the United States and the European
Union for the treatment of aHUS. Alexion is evaluating other potential
indications for Soliris and is developing four other highly innovative
biotechnology product candidates. This press release and further
information about Alexion Pharmaceuticals, Inc. can be found at: www.alexionpharma.com.
[ALXN-G]
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Alexion Pharmaceuticals, Inc.
Irving Adler, 203-271-8210
Sr.
Director, Corporate Communications
or
Media:
Makovsky
+ Company
Mark Marmur, 212-508-9670
or
Investors:
Rx
Communications
Rhonda Chiger, 917-322-2569
Source: Alexion Pharmaceuticals, Inc.
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