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Pipeline

Alexion’s global R&D organization is working to advance the most robust rare disease pipeline in the biotech industry. Our highly innovative product candidates are being evaluated 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

 
 

SBC-103 for Mucopolysaccharidosis IIIB (MPS IIIB)
Phase: Early Clinical Development

 
 

ENPP1 for Generalized Arterial Calcification of Infancy (GACI)
Phase: Preclinical

 
 

ALXN1540 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