Building a Better Tomorrow, Every Day

RESEARCH AND DEVELOPMENT

Building a Better Tomorrow, Every Day

Our innovation and expertise in rare disease are driven by a sense of urgency and a relentless quest for answers because lives are at stake. Through research and development we’re building a better tomorrow, every day.

Leading in Rare Disease R&D

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, understanding, and responding to the communities we serve.

Rare disease research and development (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.

30 YEARS OF RARE DISEASE LEADERSHIP

Our R&D innovations are fueled by our unmatched rare disease expertise, unyielding perseverance, and focus on helping more patients and their families around the world.

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LEADING IN RARE DISEASE COLLABORATION

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LEADING IN PATIENT CENTRICITY: WHY IT MATTERS

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LEADING IN RARE NEUROLOGICAL DISEASE RESEARCH

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PATIENT CENTRICITY IN ACTION

LEADING IN RARE REGULATORY PATHWAYS

LEADING IN RARE DISEASE CLINICAL TRIALS

Our Pioneering Science

Discover how our scientists’ bold translational research in complement biology led to transformational medicines for rare diseases and how we’re applying our expertise beyond complement to deliver innovations for more patients and their families.

OUR SCIENCE

Innovation Is in Our Pipeline

We continue to deepen our understanding of rare disease, building on our trailblazing science in complement inhibition and more than 3 decades of expertise in the space to help even more patients.

Our development efforts focus on the core therapeutic areas of hematology, nephrology, neurology, metabolics, and cardiology.

Today, we continue to expand into new complement indications, explore new targets beyond complement, and strengthen our clinical-stage pipeline through internal and external development opportunities in our core areas.

Shortening the Rare Disease Diagnostic Odyssey

One of the most significant challenges facing the rare disease community is the length of time it can take for a patient to receive an accurate diagnosis of their disease. As part of Alexion’s unwavering commitment to supporting people living with rare diseases and their families, we are focused on helping patients get answers faster. In partnership with scientific and patient communities and government agencies, we are working to identify diagnostic barriers and develop solutions to improve disease management, including pursuing science-driven opportunities with the greatest potential to improve and shorten the rare disease diagnostic journey.

Join the Effort

Clinical trials are a critical part of our efforts to develop innovative new medicines for patients with severe and devastating rare diseases. We have ongoing and actively recruiting clinical trials in a wide range of therapeutic areas.

See what’s in our rare disease pipeline

Learn more about our key ongoing clinical trials

Clinical Data Transparency and Data Disclosure

Alexion is indebted to patients and their families for their participation in clinical studies that help to advance treatments. As an organization, we are committed to voluntary sharing of Alexion-sponsored clinical trial data to address unmet medical/clinical needs.

Clinical study participant-level data will be shared publicly only if participant privacy is assured through methods like data de-identification, pseudonymization, or anonymization (as required by applicable law), and if such disclosure was included in the relevant study informed consent form or similar documentation. 

Qualified academic investigators may request participant-level clinical data and supporting documents (statistical analysis plan and protocol) pertaining to Alexion-sponsored studies. Further details regarding data availability and instructions for requesting information are available in our Clinical Trials Disclosure and Transparency Policy. If you would like additional information, please click the link below and fill out our medical information inquiry form.

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Our Externally Sponsored Research

Alexion is committed to collaborating with researchers on innovative projects that advance medical and scientific knowledge about our products, product candidates and therapeutic areas of interest. Alexion’s Externally Sponsored Research includes Investigator Sponsored Research and Externally Sponsored Collaborations.

EXTERNALLY SPONSORED RESEARCH

Our Discovery Partnerships

Alexion encourages communication of innovative ideas in rare diseases. Applications for support or collaboration for basic research are accepted from individuals or groups, as either concepts or protocols.

DISCOVERY PARTNERSHIPS
Joe, diagnosed with PNH at 24 years old, training for mixed martial arts
If anything, living with PNH has made me appreciate my time even more, and given me more drive to get back to doing what I love to do.”
JOE LIVING WITH PNH