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.
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.
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.
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.
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