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.

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Soliris® (eculizumab)

Approved Indications

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).


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.


Investigational Indications — Neurology and Transplant

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.


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.


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.


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.


Next Generation Portfolio





Investigational Candidates - Metabolic Disorders

Asfotase Alfa

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.


cPMP Replacement Therapy (ALXN1101)

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.


Investigational Candidates - Inflammatory Disorders

ALXN1007 Anti-Inflammatory Antibody

Antiphospholipid Syndrome (APS)


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


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.


Preclinical Candidates

Messenger RNA (mRNA) Therapeutics


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.