Alexion Joins Patient Organizations Worldwide in Recognizing Rare Disease Day 2014

  Alexion Joins Patient Organizations Worldwide in Recognizing Rare Disease
  Day 2014

    – Global Effort Reinforces Need for Education, Improved Diagnosis and
                         Treatment of Rare Diseases –

Business Wire

CHESHIRE, Conn. -- February 28, 2014

Alexion Pharmaceuticals (Nasdaq:ALXN) joins the European Organization for Rare
Diseases (EURORDIS), the National Organization for Rare Disorders (NORD) and
patient organizations worldwide in recognizing Rare Disease Day 2014, a global
effort to increase awareness of rare diseases, their profound impact on
patients and the need for improved diagnosis and treatment.

Alexion supports key objectives of EURORDIS, NORD and other patient
organizations through its mission to develop and deliver life-transforming
therapies for patients worldwide who suffer from severe and life-threatening
rare diseases.

Diagnosis and Treatment Challenges for Rare and Ultra-Rare Diseases

Many rare and ultra-rare diseases are chronic, progressive and marked by
continuing pain, severe disability and high mortality. Few physicians are
familiar with diagnosing and treating these illnesses, which frequently leads
to missed, delayed or inaccurate diagnoses even when an approved, effective
therapy is available.^1 According to global survey results published in the
current issue of The Journal of Rare Disorders (JRD), conducted on behalf of
Global Genes, patients living with rare diseases visited an average of 7.3
physicians before receiving an accurate diagnosis.^2 The survey also reveals
that 44 percent of patients surveyed believed that a slow diagnosis resulted
in delayed treatment with a negative impact on their condition.^2

“Patients with rare and ultra-rare life-threatening disorders often face long
delays in receiving an accurate diagnosis. Without an accurate diagnosis, or
access to effective treatment options, patients too often are at risk for
catastrophic medical consequences,” said Leonard Bell, M.D., Chief Executive
Officer of Alexion. “On this Rare Disease Day and every day, through our
continued commitment to breakthrough medical research, each of us at Alexion
are focused on improving the knowledge of rare diseases and developing and
delivering therapies to transform the lives of these patients worldwide.”

Delivering Life-Transforming Therapies Across the Globe

Alexion’s research and development programs are focused on highly innovative
therapies with the potential to transform the lives of patients with severe
and life-threatening ultra-rare disorders and for which there are no effective
treatment options. The company’s development programs are solely focused on:

  *Severe disorders with devastating and life-threatening medical
  *Disorders with ineffective, or no treatment options
  *Disorders that are ultra-rare and affect very small numbers of patients

Scientists at Alexion are working relentlessly to understand the underlying
causes of these diseases and to discover and develop breakthrough medicines to
treat them. To learn more about Alexion’s Research & Development programs,

To learn more about Rare Disease Day, visit for U.S.
activities and for global activities.

About Rare and Ultra-Rare Disorders

In the United States, a disease is defined as rare if it affects fewer than
650 patients per million of population.^3 The European Union definition of a
rare disease is one that affects fewer than five patients per 10,000 of
population.^4 In contrast, a disease is generally considered to be ultra-rare
if it affects fewer than 20 patients per million of population^5 (one patient
per 50,000) – and most ultra-rare diseases affect far fewer people than this.

Despite the very small numbers of patients they affect, the impact of these
rare and ultra-rare diseases on patients, their families, and society is
profound, as many of these conditions are severe, chronic and progressive,
with significant premature mortality. Patients with severe and
life-threatening ultra-rare diseases often live without hope, have no
effective treatment options and may face premature death.

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 a treatment for patients
with PNH and aHUS, two debilitating, ultra-rare and life-threatening disorders
caused by chronic uncontrolled complement activation. This press release and
further information about Alexion can be found at:



1. EURORDIS. The Voice of 12,000 Patients: Experiences and Expectations of
Rare Disease Patients on Diagnosis and Care in Europe. 2009.

2. Engel PA, Gabal S, Broback M, Boice N. Physician and patient perceptions
regarding physician training in rare diseases: the need for stronger
educational initiatives for physicians. J Rare Dis. 2013; 1(2):1-15.

3. U.S. Food and Drug Administration. Definition of Disease Prevalence for
Therapies Qualifying Under Orphan Drug Act:

OF THE COUNCIL of 16 December 1999 on orphan medicinal products and from
2011 on the application of patients’ rights in cross-border healthcare.

5. UK National Institute for Clinical Effectiveness (NICE). Citizen Council
Report on Ultra-Orphan Drugs. 2004; 27-28. Available at


Alexion Pharmaceuticals, Inc.
Irving Adler, 203-271-8210
Executive Director, Corporate Communications
Kim Diamond, 203-439-9600
Senior Director, Corporate Communications
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