At Alexion, AstraZeneca Rare Disease pioneering in science is in our DNA. We innovate on behalf of patients and families with unmet need and follow the science to deliver life-changing medicines. We do this because the challenge of rare disease is enormous, and the patient need is high. Of the 10,000 known rare diseases, 90% do not have an approved treatment.1
We have more than 30 years of rare disease leadership and 20-plus years of real-world evidence.
Today, this has resulted in 7 approved medicines across 8 rare diseases.
With one of the most robust early to late-stage research pipelines in rare disease, we aim to deliver five (5) new medicines by 2030. We are expanding our leadership in rare disease with multiple Phase III trials underway across disease areas, including rare haematology, nephrology, neurology, bone metabolism, cardiology, endocrinology and rare tumours.
1.Global Genes. Rare Disease Facts [Internet]. Aliso Viejo (CA): Global Genes; [cited 2026 Apr 30]. Available from: https://globalgenes.org/rare-disease-facts/
Alexion, AstraZeneca Rare Disease has designed and developed, in partnership with UPF Barcelona School of Management, a Master’s Degree in Medical Communication for the Pharmaceutical and Healthcare Industry which aims at preparing professionals to lead at the intersection of science, communication, and strategy.
Scan the QR code to learn about the Master’s Degree in Medical Communication for the Pharmaceutical and HealthCare Industry.
