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Pipeline

Alexion is advancing its rare disease pipeline with highly innovative product candidates in multiple therapeutic areas, including severe and life-threatening complement disorders and metabolic diseases.

Full Pipeline

Market
Registration
Advanced Clinical Development
Early Clinical
Development
Preclinical
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Soliris® (eculizumab) for Paroxysmal Nocturnal Hemoglobinuria (PNH)
Phase: Market

 
 

Soliris® (eculizumab) for Atypical Hemolytic Uremic Syndrome (aHUS)
Phase: Market

 
 

Eculizumab for Refractory Generalized Myasthenia Gravis (gMG)
Phase: Registration

 
 

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

 
 

Eculizumab for Antibody Mediated Rejection (AMR)
Phase: Advanced Clinical Development

 
 

ALXN1210 for PNH
Phase: Advanced Clinical Development

 
 

ALXN1210 for aHUS
Phase: Advanced Clinical Development

 
 

ALXN1210—Subcutaneous
Phase: Early Clinical Development

 
 

Other Complement Inhibitors
Phase: Preclinical

 
 

Strensiq® (asfotase alfa) for Hypophosphatasia (HPP)
Phase: Market

 
 

Kanuma® (sebelipase alfa) for Lysosomal Acid Lipase Deficiency (LAL-D)
Phase: Market

 
 

ALXN1101 (cPMP Replacement Therapy) for Molybdenum Cofactor Deficiency (MoCD) Type A
Phase: Advanced Clinical Development

 
 

ALXN1630 for Crigler-Najjar Syndrome (CN-1)
Phase: Preclinical

 
 

Other Metabolic Candidates
Phase: Preclinical

 
 

Other mRNA Therapies
Phase: Preclinical

 
 

Samalizumab (ALXN6000) for Solid Tumors
Phase: Early Clinical Development

 
 

Samalizumab (ALXN6000) for Acute Myeloid Leukemia (AML)
Phase: Early Clinical Development

 
 

Other Preclinical Candidates
Phase: Preclinical