Pipeline

Alexion is advancing a rare disease pipeline that builds on our fundamental strength in complement biology and focuses on our core therapeutic areas of hematology and nephrology, neurology, metabolics and FcRn . We are steadfast in our pursuit of groundbreaking innovation, and aim to redefine what it means to live with a rare disease.

Full Pipeline

Advanced Clinical Development
Early Clinical
Development
Preclinical
 

ULTOMIRIS® (ravulizumab-cwvz) IV for PNH
Phase: Advanced Clinical Development

 
 

ULTOMIRIS IV for aHUS
Phase: Advanced Clinical Development

 
 

ULTOMIRIS IV for Neuromyelitis Optica Spectrum Disorder (NMOSD)
Phase: Advanced Clinical Development

 
 

ULTOMIRIS IV for Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy (HSCT-TMA)
Phase: Advanced Clinical Development

 
 

SOLIRIS® (eculizumab) for Generalized Myasthenia Gravis (gMG)
Phase: Advanced Clinical Development

 
 

SOLIRIS for Neuromyelitis Optica Spectrum Disorder (NMOSD)
Phase: Advanced Clinical Development

 
 

ALXN1840 for Wilson Disease
Phase: Advanced Clinical Development

 
 

ULTOMIRIS Subcutaneous QW
Phase: Advanced Clinical Development

 
 

ULTOMIRIS IV for Generalized Myasthenia Gravis (gMG)
Phase: Advanced Clinical Development

 
 

ULTOMIRIS IV for Amyotrophic Lateral Sclerosis (ALS)
Phase: Early Clinical Development

 
 

ULTOMIRIS IV for Primary Progressive Multiple Sclerosis (PPMS)
Phase: Early Clinical Development

 
 

ALXN1810 Subcutaneous Q2W or Q4W
Phase: Early Clinical Development

 
 

ALXN1830 for Warm Autoimmune Hemolytic Anemia (WAIHA) and Generalized Myasthenia Gravis (gMG)
Phase: Early Clinical Development

 
 

CAEL-101
Phase: Early Clinical Development

 
 

ABY-039
Phase: Early Clinical Development

 
 

ALXN1720
Phase: Preclinical

 
 

Peptide Therapies
Phase: Preclinical

 
 

GalXCTMRNA interference (RNAi)
Phase: Preclinical

 
 

CP010
Phase: Preclinical

 
 

Additional Complement
Phase: Preclinical