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.
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.
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 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.
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.