Rare & Ultra-Rare Diseases
Alexion is focused on providing treatments for patients with rare and ultra-rare diseases,
particularly those that profoundly affect a person's survival and quality of life, and for
which there are few, if any, effective treatment options. According to the European Organization
for Rare Diseases (EURORDIS), a disease or disorder is defined as rare when it affects fewer than
500 in one million of population (Orphan Drug Regulation 141/2000). An ultra-rare disease is generally
considered one that affects fewer than 20 patients per one million of population — and most ultra-rare diseases affect far fewer people.
The impact of rare and ultra-rare diseases on patients and their families can be profound. Many of these
disorders are severe, chronic and progressive, and are marked by pain, disability and high mortality rates.
The seriousness of rare diseases is often compounded by a lack of scientific knowledge, leading to missed diagnoses,
misdiagnoses and sub-optimal treatment. Cruelly, many of these diseases predominantly strike infants and children.
Alexion has developed exceptional scientific and operational expertise in developing therapies for patients
with rare and ultra-rare diseases. While there are many scientific, regulatory and operational hurdles unique to
ultra-orphan therapies, Alexion will continue to explore potential treatments for patients with additional rare disorders –
a commitment reflected in our research
and development programs. This commitment goes beyond developing new medicines
and includes working with physicians, governments and payors to pursue our objective of access to therapies
for all patients who can benefit from them.