Development Programs

Alexion’s development programs focus on four core therapeutic areas: hematology and nephrology, neurology, metabolics and FcRn. We are leveraging our global leadership in complement biology to expand eculizumab into new indications within these areas, while driving continued innovation with ravulizumab-cwvz, our long-acting C5 inhibitor, ALXN1840 for Wilson disease and ALXN1830 for rare IgG-mediated diseases. We are also seeking business development opportunities to strengthen our clinical-stage pipeline in the four core areas.

Learn more about our current development programs below.

ULTOMIRIS® (ravulizumab-cwvz) IV for Paroxysmal Nocturnal Hemoglobinuria (PNH)  |  See Clinical Trials

ULTOMIRIS, a long-acting C5 inhibitor, was approved for the treatment of adults with PNH, a severe and 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). A Phase 3 study in children and adolescents under the age of 18 who have PNH is currently underway.

ULTOMIRIS IV for Atypical Hemolytic Uremic Syndrome (aHUS)  |  See Clinical Trials

Alexion is evaluating ULTOMIRISadministered intravenously every eight weeks in a Phase 3 trial of complement inhibitor treatment-naive adolescent and adult patients with aHUS, a genetic, chronic, ultra-rare disease associated with vital organ failure and premature death. The company has also initiated a Phase 3 trial in pediatric patients with aHUS.

ULTOMIRIS for Subcutaneous Use |  See Clinical Trials

Initial pharmacokinetic and tolerability data from a Phase I study in healthy volunteers support progressing the development of a subcutaneous formulation of ULTOMIRIS. Based on discussions with regulators, Alexion plans to initiate a single, PK-based Phase 3 study of ULTOMIRIS delivered subcutaneously once per week to support registration in PNH and aHUS in late 2018. A Phase 1 study of ULTOMIRISco-administered with Halozyme's ENHANZE® drug-delivery technology, PH20, is underway. Pending co-formulation data, this subcutaneous formulation will be called ALXN1810 and is being evaluated for a dosing interval of once every two weeks or once per month.

ALXN1840 for Wilson Disease |  See Clinical Trials

Alexion is evaluating ALXN1840 (bis-choline tetrathiomolybdate) in a Phase 3 clinical trial for the treatment of Wilson disease, a rare genetic disorder with devastating hepatic and neurological consequences for patients. ALXN1840 is novel oral copper-protein binding agent with a unique mechanism of action and ability to access and bind to serum copper and promote its removal from the liver.

ALXN1830 for Warm Autoimmune Hemolytic Anemia (WAIHA) and Generalized Myasthenia Gravis (gMG) |  See Clinical Trials

ALXN1830 is a humanized monoclonal antibody that inhibits the interaction of neonatal Fc receptor (FcRn) with Immunoglobulin G (IgG) and IgG immune complexes and has the potential to improve treatment in a number of rare IgG-mediated diseases. Alexion plans to initiate two Phase 2/3 trials of ALXN1830 (SYNT001) in late 2019 or early 2020 - one in warm autoimmune hemolytic anemia (WAIHA) and one in gMG.

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


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