Rare Disease

Transforming the lives of people affected by rare diseases

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Our commitment

Our mission is to transform the lives of people affected by rare diseases By understanding patients’ unique needs, we can research and develop innovative medicines, support access and advocate for the rare disease community.

We believe it is our responsibility to listen to, understand, and change the lives of patients and those who work tirelessly to help them. Our innovation begins with understanding people living with rare diseases, which fuels all of our efforts.

Rare diseases by the numbers

400 million

people around the world are living with a rare disease1


rare diseases are known to exist today, but more than 90% do not have an approved treatment option2,3

5+ years

to accurate diagnosis, a path that includes multiple doctors, specialists and misdiagnoses4

Our strategy in Rare Disease

Alexion’s pioneering legacy in rare diseases is rooted in being the first to translate the complex biology of the complement system into transformative medicines. By driving innovative research and development (R&D) across new disease targets and modalities, we have diversified our pipeline into additional rare diseases over the last several years. Today, as part of AstraZeneca, we are building bridges across our scientific platforms with a focus on bringing more innovative medicines to people worldwide.

Our rare disease strategy focuses on three core priorities:

Our R&D approach

For 30 years, Alexion’s pioneering science has led to the development of transformational medicines for rare diseases, and we continue to lead the way today. Our success is tied to listening, to understanding, and responding to the communities we serve.

Rare disease R&D presents unique challenges that require continuous innovation to deliver meaningful medicines for patients. Driven by our patient-centric approach, we pave the way by tailoring elements of the R&D process and developing new tools and methods to gain a deeper understanding of many rare conditions and the needs of the people living with them.

Our people

We invest in and value people who believe in the importance of our purpose and understand what it takes to deliver on it. In everything we do, we are empowered and committed to speak up and perform at our personal best to accelerate our collective impact for people living with rare diseases. Our culture is rooted in integrity, inclusiveness, and our dedication to joining and supporting the communities in which we live and work.

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.

Marc Dunoyer, Chief Executive Officer, Alexion and Chief Strategy Officer, AstraZeneca

Join our team

We are innovators, leaders and collaborators. And at the heart of everything we do is an intense desire to pursue the toughest challenges and truly change the world for people living with rare and devastating diseases.

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

Advancing our pipeline every day

We’re building on our fundamental understanding of complement biology and advancing on our core therapy areas of haematology, nephrology, neurology, metabolics, and cardiology. We continue to evolve into new areas where there is a great unmet need and opportunity to help people living with rare diseases and their families fully live their best lives.

Phase III/LCM Projects: refers to assets that are pivotal in Phase II/III, or that have been submitted for regulatory approval, and may include assets that are now launched in one or more major markets (removed when launched in all applicable major markets).


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: Z4-62042
Date of preparation: February 2024