Ultra-Rare Diseases

Alexion is focused on providing life-transforming treatments to patients with severe and life-threatening rare diseases for which there are no effective treatment options.

The impact of these diseases on patients, their families, and society is profound as many are severe, chronic and progressive, with high mortality rates. Ultra-rare diseases often present unique public health challenges. Typically, few researchers or companies explore such diseases. It is also highly likely that very few physicians are familiar with diagnosing and treating these conditions, leading to missed, delayed or inaccurate diagnosis and thereby delaying treatment, even when an approved, effective therapy is available.

When developing treatments for patients with rare and ultra-rare diseases, often there are no pre-existing regulatory pathways or prior controlled studies. Alexion is one of the few companies in this field with a proven track record in drug discovery, regulatory affairs, clinical trial design and recruitment, and manufacturing.

Our approach to serving patients is driven by education and a passion for understanding and meeting the unique needs of patients and families suffering from rare and ultra-rare diseases.

What is an Ultra-Rare Disease?

1. World Health Organization’s Diabetes Fact Sheet, accessed here.
2. US and World Population Clock, accessed here.
3. US Food and Drug Administration’s Definition of Disease Prevalence for Therapies Qualifying Under Orphan Drug Act, accessed here.
4. Definition from the UK National Institute for Clinical Effectiveness (NICE). 2004. Citizen Council Report on Ultra-Orphan Drugs. Available here as defined in the following documents: Wales Medicines Strategy Group (AWMSG); Recommendations for a Belgian Plan for Rare Diseases; the EMINET Report commissioned by the European Commission’s Directorate General Enterprise and Industry, the European Union Committee of Experts on Rare Diseases’ (EUCERD).