Rare Disease

Transforming the lives of people affected by rare diseases and devastating conditions

Our mission is to transform the lives of people affected by rare diseases and devastating conditions. 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.

Unmet medical need and world market

400 million

people around the world are affected by a rare disease, half of whom are children.


rare diseases are known to exist today but only 5% have treatments.

3 in 10 children

with a rare disease don’t live to see their fifth birthday.

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

Unlocking the potential of the complement system

The dysregulation of the complement system, an essential part of the immune system, is a key driver of many devastating diseases. Targeting and inhibiting the complement system before it can trigger tissue damage or destruction can help restore balance. We are committed to continue unlocking the potential of the complement system, to discover new life-changing therapies for even more patients.

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 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 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.

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.


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Veeva ID: Z4-42381
Date of preparation: March 2022