Pipeline

We’re building on our fundamental understanding of complement biology and advancing on our core therapeutic areas of hematology, nephrology, neurology, metabolics, and cardiology. We continue to evolve into new areas where there is great unmet need and opportunity to help people living with rare diseases and their families fully live their best lives.

Full Pipeline

Phase 3
Phase 1/2
Preclinical
 

SOLIRIS® (eculizumab) for Generalized Myasthenia Gravis (gMG)
Phase: 3

 
 

SOLIRIS for Neuromyelitis Optica Spectrum Disorder (NMOSD)
Phase: 3

 
 

ULTOMIRIS® (ravulizumab-cwvz) IV for PNH
Phase: 3

 
 

ULTOMIRIS IV for aHUS
Phase: 3

 
 

ULTOMIRIS Subcutaneous QW
Phase: 3

 
 

ULTOMIRIS IV for Generalized Myasthenia Gravis (gMG)
Phase: 3

 
 

ULTOMIRIS IV for Neuromyelitis Optica Spectrum Disorder (NMOSD)
Phase: 3

 
 

ULTOMIRIS IV for Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy (HSCT-TMA)
Phase: 3

 
 

ULTOMIRIS IV for Amyotrophic Lateral Sclerosis (ALS)
Phase: 3

 
 

ALXN1840(WTX101) for Wilson Disease
Phase: 3

 
 

CAEL-101
Phase: 3

 
 

AG10
Phase: 3

 
 

Elamipretide1
Phase: 3

 
 

ALXN1830 (SYNT001) for Warm Autoimmune Hemolytic Anemia (WAIHA) and Generalized Myasthenia Gravis (gMG)
Phase: 1/2

 
 

Danicopan (ACH-4471) & ACH-52282
Phase: 1/2

 
 

ULTOMIRIS IV for Primary Progressive Multiple Sclerosis (PPMS)
Phase: 1/2

 
 

ALXN1810 Subcutaneous Q2W or Q4W
Phase: 1/2

 
 

ABY-039
Phase: 1/2

 
 

ALXN1720
Phase: 1/2

 
 

Peptide Therapies
Phase: Preclinical

 
 

GalXCTMRNA interference (RNAi)
Phase: Preclinical

 
 

CP010
Phase: Preclinical

 
 

Immune Pharma - anti-eotaxin-1 antibody3
Phase: Preclinical

 
 

Additional Complement
Phase: Preclinical

 
 
 

  1. Option to co-develop and commercialize
  2. Subject to transaction closure
  3. Subject to completion of bankruptcy proceedings