Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) joins the European Organization for Rare Diseases (EURORDIS), the National Organization for Rare Disorders (NORD) and families, governments, companies and medical communities around the world in observing the fourth annual Rare Disease Day today.
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 experts," said Peter L. Saltonstall, President and CEO of NORD. "On Rare Disease Day 2011, millions of patients and their families will share their stories to shine a light on rare diseases and help educate and inform the public."
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 day," said Leonard Bell, M.D., Chief Executive Officer of Alexion. "We are gratified to see how far we've come in changing the landscape of how PNH is diagnosed, treated and managed. But much work remains to help patients suffering from rare diseases, and we are accelerating our programs to develop life-transforming therapies for other rare and severe disorders."
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 group in Cambridge, Massachusetts to accelerate the development of an expanded portfolio of innovative preclinical compounds. One such compound is an investigational treatment for newborn children with molybdenum cofactor deficiency (MoCD) Type A, a catastrophic, ultra-rare genetic disorder that strikes newborns and currently has no treatment options.