Global Event Aims to Improve Diagnosis, Treatment, Access and Support for Patients and Families Facing Rare, Devastating Diseases
In the U.S., a rare disease is defined as one that affects fewer than 200,000 patients. Many diseases are considered ultra-rare, generally thought of as affecting fewer than 20 patients per one million of the population, or approximately 6,000 or fewer patients in the U.S. The goal of Rare Disease Day is to draw attention to these diseases as an important public health issue with unique challenges. This year's theme, "rare but equal," spotlights the healthcare inequalities that often exist with rare diseases. Specific objectives include equal access to health care and social services; equal access to orphan drugs and treatments; and equal access to basic social rights such as education and employment.
"Challenges faced by patients with rare diseases include delayed
diagnosis, few treatment options, and difficulty finding medical
As a company dedicated to developing innovative therapies for patients with rare, devastating disorders, Alexion shares the mission of Rare Disease Day to elevate research, hope and equality for patients. 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. Today, the company is investigating the potential of Soliris in other rare and ultra-rare disorders, including atypical Hemolytic Uremic Syndrome (aHUS), an ultra-rare genetic disorder, and acute humoral rejection (AHR) in patients undergoing kidney transplant.
"Rare Disease Day brings much-needed attention to the efforts that many
advocates, physicians, policymakers and companies are engaged in every
Rare Disease Day 2011 coincides with The NASDAQ Stock Market's
recognition of Alexion's 15th anniversary as a listed company at this
morning's Market Open ceremony. With the goal of breaking new ground in
rare diseases, Alexion recently established a translational medicine
To learn more about Alexion's research and development programs, access initiatives, and mission and vision, 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.
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. Approximately 10 percent of all patients first develop symptoms at 21 years of age or younger. 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. It is estimated that approximately one-third of patients with PNH do not survive more than five years from the time of diagnosis. PNH has been identified more commonly among patients with disorders of the bone marrow, including aplastic anemia (AA) and myelodysplastic syndromes (MDS). In patients with thrombosis of unknown origin, PNH may be an underlying cause. More information on PNH is available at www.pnhsource.com.
Soliris is a first-in-class terminal complement inhibitor developed from
the laboratory through regulatory approval and commercialization by
Alexion. Soliris has been approved in the U.S.,
Important Safety Information
Soliris is generally well tolerated in patients with PNH. The most frequent adverse events observed in clinical studies of patients with PNH were headache, nasopharyngitis (runny nose), back pain and nausea. Treatment with Soliris should not alter anticoagulant management because the effect of withdrawal of anticoagulant therapy during Soliris treatment has not been established.
The U.S. product label for Soliris also includes a boxed warning: "Soliris increases the risk of meningococcal infections. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early. Vaccinate patients with a meningococcal vaccine at least two weeks prior to receiving the first dose of Soliris; revaccinate according to current medical guidelines for vaccine use. Monitor patients for early signs of meningococcal infections, evaluate immediately if infection is suspected, and treat with antibiotics if necessary." During PNH clinical studies, two out of 196 vaccinated PNH patients treated with Soliris experienced a serious meningococcal infection. Prior to beginning Soliris therapy, all patients and their prescribing physicians are encouraged to enroll in the PNH Registry, which is part of a special risk-management program that involves initial and continuing education and long-term monitoring for detection of new safety findings.
Safe Harbor Statement
This news release contains forward-looking statements, including
statements related to potential health and medical benefits of Soliris
(eculizumab) for the treatment of patients with PNH, and Alexion's plans
for developing other therapies and for developing Soliris for other
disorders. 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 its current or potential new indications, and a variety of other
risks set forth from time to time in Alexion's filings with the
Senior Director, Corporate Communications and Public Policy
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