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Pipeline

Alexion’s global R&D organization is working to advance the most robust pipeline in the Company’s history. Our pipeline programs are highly focused and span several therapeutic areas including hematology, nephrology, transplant, neurology, metabolic disorders and inflammatory disorders. Today, we have programs underway with multiple highly innovative therapeutic candidates, including eculizumab, that have the potential to become first-in-class therapies for patients with severe and life-threatening disorders.

  • Preclinical
  • Early Clinical Development
  • Advanced Clinical Development
  • Registration Filings
  • Market

Complement Inhibitor Portfolio

Soliris® (eculizumab) for Paroxysmal Nocturnal Hemoglobinuria (PNH)

 
 

Soliris is approved for the treatment of PNH in nearly 50 countries, including the United States, European Union and Japan. PNH is an ultra-rare blood disorder in which chronic, uncontrolled activation of complement, a component of the normal immune system, results in hemolysis (destruction of the patient's red blood cells).

 

Soliris® (eculizumab) for Atypical Hemolytic Uremic Syndrome (aHUS)

 
 

Soliris is approved for the treatment of aHUS in nearly 40 countries, including the United States, European Union and Japan. aHUS is a chronic, ultra-rare, and life-threatening disease in which a lifelong and permanent genetic deficiency in one or more complement regulatory genes causes chronic uncontrolled complement activation, resulting in complement-mediated thrombotic microangiopathy (TMA), the formation of blood clots in small blood vessels throughout the body.

 

Eculizumab for Refractory Myasthenia Gravis (MG)

 
 

Eculizumab is being investigated in refractory MG, a debilitating and sometimes life-threatening neurological disorder in which uncontrolled complement activation leads to destruction and inflammation at the neuromuscular junction, resulting in severe muscle weakness.

 

Eculizumab for Relapsing Neuromyelitis Optica (NMO)

 
 

Eculizumab is being investigated as a treatment for relapsing NMO, a devastating, life-threatening, ultra-rare neurological disease that can lead to severe weakness, paralysis, respiratory failure, loss of bowel and bladder function, blindness and premature death.

 

Eculizumab for Delayed Graft Function (DGF)

 
 

Eculizumab is being evaluated in DGF, an early and serious complication of organ transplantation in which a transplanted kidney, known as a graft, does not function normally immediately following transplantation. Patients experiencing DGF after a kidney transplant require dialysis in order to survive, and the condition can be life-threatening due to the risk of losing the transplanted organ.

 

Eculizumab for Antibody Mediated Rejection (AMR)

 
 

Eculizumab is being investigated in patients undergoing kidney transplant who are at elevated risk of AMR, a severe and potentially life-threatening condition that can lead to kidney allograft damage, resulting in rapid loss of function and possible loss of the transplanted kidney.

 

ALXN1007 for Gastrointestinal Graft-versus-Host Disease (GI-GVHD)

 
 

ALXN1007 is a novel anti-inflammatory antibody being investigated in graft-versus-host disease involving the lower gastrointestinal tract (GI-GVHD), a severe and potentially life-threatening autoimmune disorder.

 

ALXN1007 for Antiphospholipid Syndrome (APS)

 
 

ALXN1007 is a novel anti-inflammatory antibody being investigated in APS, a severe and potentially life-threatening autoimmune disorder.

 

ALXN1210

 
 

ALXN5500

 
 

Metabolic Portfolio

StrensiqTM (asfotase alfa) for Hypophosphatasia (HPP)

 
 

Asfotase alfa is a first-in-class enzyme replacement therapy being investigated as a potential treatment for patients with HPP, a genetic, chronic and progressive ultra-rare metabolic disease characterized by defective bone mineralization that can lead to destruction and deformity of bones, profound muscle weakness, seizures, respiratory failure and premature death.

 

KanumaTM (sebelipase alfa) for Lysosomal Acid Lipase Deficiency (LAL-D)

 
 

Sebelipase alfa is a recombinant form of the human LAL enzyme being developed as an enzyme replacement therapy for patients with LAL-D, a genetic, chronic and rare metabolic disease associated with multi-systemic organ damage including hepatic fibrosis, cirrhosis, liver failure, accelerated atherosclerosis, cardiovascular disease, and other devastating consequences.

 

ALXN1101 (cPMP Replacement Therapy) for Molybdenum Cofactor Deficiency (MoCD) Type A

 
 

cPMP (cyclic pyranopterin monophosphate) is an enzyme co-factor replacement therapy for the treatment of patients with molybdenum cofactor deficiency (MoCD) type A, a severe, life-threatening, ultra-rare, genetic metabolic disorder that causes catastrophic and irreversible neurologic damage within the first weeks of life.

 

SBC-103 for Mucopolysaccharidoses IIIB (MPS IIIB)

 
 

SBC-103 is an enzyme replacement therapy being investigated as a treatment for patients with MPS IIIB, a rare, devastating and life-threatening disease that typically presents in children during the first few years of life and causes progressive and severe cognitive decline, behavioral problems, speech loss, increasing loss of mobility, and premature death.

 

Preclinical Candidates

mRNA Therapies

 
 

Alexion is partnering with Moderna Therapeutics on the discovery and development of messenger RNA Therapeutics™ to treat rare diseases.

 

Asfotase alfa for Neurofibromas Type 1 (NF1)

 
 

Asfotase alfa is a first-in-class enzyme replacement therapy being investigated as a potential treatment for patients with Neurofibromatosis Type 1, or NF-1, a devastating disease in which excess pyrophosphate (PPi) may play a role.

 

SBC-105 for Generalized Arterial Calcification of Infancy (GACI)

 
 

SBC-105 is being investigated as a potential treatment for patients with generalized arterial calcification of infancy (GACI), a rare, genetic, and life-threatening disease that typically presents in the first month of life. SBC-105 is also being investigated for other rare disorders of calcification.

 

Other Complement Inhibitors

 

Alexion is partnering with Moderna Therapeutics on the discovery and development of messenger RNA Therapeutics™ to treat rare diseases.

 

Other Preclinical Candidates

 
 

Alexion’s Research & Development pipeline also includes several additional preclinical programs across various therapeutic areas.