Ultra-Rare Diseases
Alexion is focused on providing treatments for patients with ultra-rare and serious diseases,
particularly those disorders which profoundly affect a person's survival and quality of life,
and for which there are few, if any, effective treatment options. Through the company's
experience with Soliris
and PNH, Alexion
has developed exceptional scientific and operational expertise in the field of ultra-rare
disease, generally defined as conditions that affect fewer than 20 people per one
million of population.
The impact of ultra-rare disease 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 ultra-rare diseases is often compounded by
a lack of scientific knowledge, leading to missed diagnoses, mis-diagnoses and sub-optimal
treatment. Cruelly, many of these diseases predominantly strike infants and children.
While there are many scientific, regulatory and operational hurdles associated with
developing therapies for patients with rare and ultra-rare diseases, Alexion will continue
to explore potential treatments for patients with other rare disorders – a commitment
reflected in our research
and development programs. Our 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.