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Our Research

Patients are at the forefront of our R&D strategy. We understand that patients suffering from severe, life-threatening rare diseases often have no effective treatment options and little hope. Our R&D teams focus exclusively on these underserved patient populations, and work closely with the medical community to understand the pathophysiology, natural history and clinical consequences of these devastating diseases, so that we can identify and investigate potential breakthrough therapies.

Today, Alexion is developing the most robust rare disease pipeline in the biotech industry, with highly innovative product candidates in multiple therapeutic areas:

  • As the global leader in complement inhibition, Alexion is strengthening and broadening its portfolio of complement inhibitors across diverse platforms, including investigating Soliris (eculizumab) as a treatment for patients with life-threatening rare disorders in the areas of neurology and transplant. Our lead next generation Soliris molecule, ALXN1210, is being evaluated as a treatment for PNH. Alexion is also investigating ALXN1007, a novel anti-inflammatory antibody being studied in Gastrointestinal Graft-versus-Host Disease (GI-GVHD) and Antiphospholipid Syndrome (APS).  
  • Alexion’s metabolic development programs include ALXN1101, an investigational cPMP replacement therapy being evaluated for patients with molybdenum cofactor deficiency (MoCD) Type A, and SBC-103, an investigational enzyme replacement therapy being evaluated for patients with mucopolysaccharidosis IIIB (MPS IIIB).
  • In addition, Alexion is investigating a preclinical pipeline of more than 30 diverse programs across a range of therapeutic modalities.