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Development Programs

Alexion has a broad range of development programs under way to bring much-needed therapies to patients suffering from severe and devastating rare diseases. Our current programs are focused on complement-mediated disorders and metabolic disorders that currently have few, if any, effective treatments.

Complement Programs

Eculizumab for Refractory Generalized Myasthenia Gravis (gMG)  |  See Clinical Trials

Alexion has submitted marketing applications in the U.S. and EU for Soliris for the treatment of patients with refractory gMG who are anti-acetylcholine receptor (AChR) antibody-positive. Refractory gMG patients who are AChR antibody-positive represent an ultra-rare segment of patients with MG—a debilitating, complement-mediated neuromuscular disease—who experience severe morbidities despite currently available MG therapies.

Eculizumab for Neuromyelitis Optica Spectrum Disorder (NMOSD)  |  See Clinical Trials

Neuromyelitis optica spectrum disorder (NMOSD) is a life-threatening, ultra-rare autoimmune neurological disorder in which complement activation by antibodies against aquaporin-4 on astrocyte cell surfaces causes damage in the central nervous system, including the spinal cord and optic nerve.3-5 The disease leads to severe weakness, paralysis, respiratory failure, loss of bowel and bladder function, blindness and premature death. Most patients experience an unpredictable, relapsing course of disease where each individual attack adds to cumulative neurologic disability.6-10

Alexion is currently enrolling patients in the PREVENT (Prevention of Relapses and EValuation of Eculizumab in NMO Treatment) study to evaluate the safety and efficacy of eculizumab as a potential treatment for patients with relapsing NMOSD.

Eculizumab for Antibody-Mediated Rejection (AMR)  |  See Clinical Trials

Acute antibody-mediated rejection (AMR) is a severe and potentially life-threatening condition that can lead to severe kidney allograft damage, resulting in rapid loss of function and possible loss of the transplanted kidney.1 Research suggests that uncontrolled complement activation, triggered by the binding of donor-specific antibodies (DSAs) to target proteins in the donor kidney, may be the primary cause of acute AMR in kidney transplant recipients.1,2

Currently, there are no approved therapies for the prevention or treatment of AMR. Alexion is investigating eculizumab in patients receiving kidney transplants who are at elevated risk of AMR.

ALXN1210 for Paroxysmal Nocturnal Hemoglobinuria (PNH) and Atypical Hemolytic Uremic Syndrome (aHUS)  |  See Clinical Trials

ALXN1210 is a highly innovative, longer-acting anti-C5 antibody being evaluated by Alexion in Phase 3 trials in patients with PNH and aHUS. 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). aHUS is a genetic, chronic, ultra-rare disease associated with vital organ failure and premature death.

Metabolic Programs

ALXN1101 (cPMP Replacement Therapy) for Molybdenum Cofactor Deficiency (MoCD) Type A  |  See Clinical Trials

MoCD Type A is a severe, life-threatening, ultra-rare genetic metabolic disease affecting newborns in which a genetic deficiency of cyclic pyranopterin monophosphate (cPMP) results in the inability of the body to form an essential cofactor called molybdenum cofactor. MoCD Type A causes catastrophic and irreversible neurologic damage within the first weeks of life.11

Alexion is advancing the development of ALXN1101, a cPMP replacement therapy for the treatment of patients with MoCD Type A.

Our Clinical Trials

Alexion is investigating new therapies that have the potential to transform patients' lives. Learn more about some of our key clinical studies.

Alexion Clinical Trials


  1. Takemoto SK, Zeevi A, Feng S, et al. National conference to assess antibody-mediated rejection in solid organ transplantation. Am J Transplant. 2004; 4(7):1033-41.
  2. Collins AB, Schneeberger EE, Pascual MA, et al. Complement activation in acute humoral renal allograft rejection: diagnostic significance of C4d deposits in peritubular capillaries. J Am Soc Nephrol. 1999;10(10):2208-14.
  3. Jarius S, Aboul-Enein, Waters P, et al. Antibody to AQP4 in the long term course of neuromyelitis optica. Brain. 2008;131:3072-80.
  4. Hinson SR, Romero MF, Popescu BFG, et al. Molecular outcomes of neuromyelitis optica (NMO)-IgG binding to aquaporin-4 in astrocytes. Proc Nat Acad Sci 2012;109(4):1245-50.
  5. Hinson SR, Pittock SJ, Lucchinetti CF, et al. Pathogenic potential of IgG binding to water channel extracellular domain in neuromyelitis optica. Neurology 2007;69:2221-31.
  6. Wingerchuk DM, Lennon VA, Lucchinetti CF, Pittock SJ, Weinshenker BG. The spectrum of neuromyelitis optica. Lancet Neuro. 2007;6(9):805-15.
  7. Wingerchuk DM. Diagnosis and treatment of neuromyelitis optica. Neurologis. 2007;13(1):2-11.
  8. Wingerchuk DM, Weinshenker BG. Neuromyelitis optica (Devics Syndrome). Chapter 26, Handbook of Clinical Neurology, Vol. 122 (3rd series) Multiple Sclerosis and Related Disorders, Elsevier, 2014
  9. Tuzun E, Kurtuncu M, Turkoglu R, et al. Enhanced complement consumption in neuromyelitis optica and Behcet’s disease patients. J Neuroimmunol 2011;233(1-2):211-5.
  10. Kuroda H, Fujihara K, Takano R, et al. Increase of complement fragment C5a in cerebrospinal fluid during exacerbation of neuromyelitis optica. J Neuroimmunol 2013;254(1-2):178-82.
  11. Vijayakumar_Pediatric Neurology_45_2011; Per, Brain Dev.2005; Serrano, Pediatr Radiol, 2007.