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
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.
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:
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.
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.
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.
Ultomiris met primary endpoint in CHAMPION-NMOSD Phase III trial in adults with neuromyelitis optica spectrum disorder
Ultomiris demonstrated sustained improvements in functional activities and quality of life in adults with generalised myasthenia gravis through 60 weeks
AstraZeneca and Neurimmune close exclusive global collaboration and licence agreement to develop and commercialise NI006
AstraZeneca and Neurimmune sign exclusive global collaboration and licence agreement to develop and commercialise NI006
Veeva ID: Z4-42381
Date of preparation: March 2022