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Rare Disease
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Driven by a Mission to Change What It Means to Live with a Rare Disease
Built on a legacy of turning pioneering science into transformative treatments, we listen to and partner with the rare disease ecosystem to help improve outcomes for more people impacted by rare diseases across the globe.
Making an Impact for the Rare Disease Community
Developing breakthrough therapies for rare diseases is only possible because of the tireless efforts of our global community of researchers and scientists, advocates and caregivers, and most important, patients.
We have the potential to impact the lives of even more people around the world.
Rare diseases by the numbers
400 million
10,000
5+ years
Our Philosophy
Every day, we're inspired to think differently and follow the science to transform the lives of people around the world living with a rare disease. These five pillars continue to guide our decisions.
Pioneers in Science
Advancing the next wave of rare disease innovation to revolutionize care and explore potential cures for patients.
Patient-Focused Innovators
Cultivating deep connections with rare disease patients and caregivers to integrate their insights and lived experience into everything we do.
Advancing our Global Footprint
Expanding the reach of rare disease research, knowledge and access to treatments around the globe.
Championing Health Equity
Taking bold steps to speed access to diagnosis and equitable care for the benefit of patients and society
Purpose-Driven People
Uniting our teams, driven by our commitment to pioneer life-changing outcomes for the rare disease community and for the health systems we operate in.
Rare disease news and stories
Trailblazers in Rare Disease
Our core identity as rare disease pioneers instills a mindset that is also rare – we courageously embrace uncertainty and follow the science to make a difference for patients. This demands a nimble and collaborative approach:
- We empower colleagues to find the right solution for a given disease without limitations on specific platforms or technology
- We design research frameworks to ensure trials with small samples are both scientifically rigorous and clinically meaningful
- We infuse patient insights into every stage of R&D process to ensure trials and medicines represent patients’ unique needs
Our people
We are united by a commitment to push the boundaries of science to improve the lives of rare disease patients. We are driven to advance this impactful work alongside talented and passionate colleagues who share our values of improving the health of people, society and the planet, so that every person has the opportunity to live their healthiest life.
I am delighted to work with Alexion colleagues to discover, develop and deliver medicines that change the lives of people suffering from rare diseases. We also look forward to applying Alexion’s complement biology platform across areas of AstraZeneca’s broader early-stage pipeline, and to making our rare disease medicines available to patients in many countries where AstraZeneca has a strong presence.
Join our team
We are relentless in our determination to do more and better for the rare disease community – and that starts with our people. We are continuously growing our skills, capabilities and behaviors through an expanding suite of professional development programs and opportunities, empowering our people to lead, innovate and take bold steps forward.
Our medicines
We cannot provide detailed information about our prescription medicines on this website, in compliance with regulations.
Our medicines are approved in individual countries for specific uses and the information we provide for patients is governed by local regulations. In some cases, health care professionals and patients can visit local AstraZeneca websites to find out more about our medicines. Please note that in some countries we are not allowed to provide very much, or sometimes any, information on our prescription medicines so you should seek alternative trustworthy sources. Always ask a healthcare professional for advice about medicines.
Our pipeline for Rare Disease
As leaders in rare disease, we draw inspiration from our history of delivering scientific firsts that have changed the course of rare disease R&D.
Our legacy in rare diseases is rooted in being the first to translate the complex biology into transformative medicines.
Today, we continue innovating with our pioneering spirit across diverse areas of unmet need — where scientific progress has been absent or limited — advancing first- and/or best-in-class medicines and new modalities.
References
1. Rare disease facts [Internet]. 2024 [cited 2024 Jan 17]. Available from: http://globalgenes.org/rare-disease-facts/
2. Fermaglich LJ, Miller KL. A comprehensive study of the rare diseases and conditions targeted by orphan drug designations and approvals over the Forty Years of the Orphan Drug Act. Orphanet Journal of Rare Diseases. 2023 Jun 23;18(1). doi:10.1186/s13023-023-02790-7
3. About [Internet]. U.S. Department of Health and Human Services; [cited 2024 Jan 17]. Available from: http://rarediseases.info.nih.gov/about
4. Barriers to Rare Disease Diagnosis, Care and Treatment in the U.S.: A 30 Year Comparative Analysis. National Organization for Rare Disorders; 2020 Nov [cited 2023 Feb 8]. Available from: http://rarediseases.org/wp-content/uploads/2020/11/NRD-2088-Barriers-30-Yr-Survey-Report_FNL-2.pdf
Veeva ID: Z4-78880
Date of preparation: November 2025