Last $177.78 USD
Change Today -2.58 / -1.43%
Volume 1.6M
As of 8:10 PM 01/28/15 All times are local (Market data is delayed by at least 15 minutes).
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Company Description

Contact Info

352 Knotter Drive

Cheshire, CT 06410

United States

Phone: 203-272-2596

Fax: 203-271-8198

Alexion Pharmaceuticals, Inc., a biopharmaceutical company, focuses on developing and commercializing life-transforming therapeutic products for serving patients with severe and ultra-rare disorders. Products and Development Programs The company’s product development programs focuses on life transforming therapeutics for severe and life-threatening ultra-rare diseases. Eculizumab is a humanized antibody known as a C5 terminal complement inhibitor (C5 Inhibitor), which is designed to selectively block the production of inflammation-causing proteins of the complement cascade. Marketed Products Paroxysmal Nocturnal Hemoglobinuria (PNH) Soliris is the therapy approved for the treatment of patients with PNH, a debilitating and life-threatening ultra-rare blood disorder in which an acquired genetic deficiency causes uncontrolled complement activation which leads to life-threatening complications. The company works with researchers to expand the base of knowledge in PNH and the utility of Soliris to treat patients with PNH. The company is sponsoring a multinational registry to gather information regarding the natural history of patients with PNH and the longer term outcomes during Soliris treatment. Atypical Hemolytic Uremic Syndrome (aHUS) aHUS is a chronic and life-threatening ultra-rare genetic disease in which uncontrolled complement activation causes blood clots in small blood vessels throughout the body (thrombotic microangiopathy, or TMA) to kidney failure, stroke, heart attack and death. The company has completed enrollment in a prospective open-label trial in adults with aHUS and, separately, enrollment has been completed in a prospective trial of pediatric patients with aHUS. Clinical Development Program Soliris (eculizumab) Nephrology Acute Antibody Mediated Rejection (AMR) in Presensitized Kidney Transplant Patients: AMR is the term used to describe a type of transplant rejection that occurs when the recipient has antibodies to the donor organ. Delayed Kidney Transplant Graft Function: The company has been granted orphan drug designation from the U.S. Food and Drug Administration (FDA) and received a positive opinion on orphan drug designation for eculizumab in DGF from the Commission on Orphan Medical Products (COMP). Shiga-toxin producing E. coli Hemolytic Uremic Syndrome (STEC-HUS): The company is obtaining and analyzing additional control clinical outcome data from an epidemiologic study in approximately 400 STEC-HUS patients. Neurology Neuromyelitis Optica (NMO): NMO is a severe and ultra-rare autoimmune disease of the central nervous system (CNS) that primarily affects the optic nerves and spinal cord. Myasthenia Gravis (MG): MG is an ultra-rare autoimmune syndrome characterized by complement activation and the subsequent failure of neuromuscular transmission. The company has completed collaborations with investigators on the design of a Phase III trial to evaluate eculizumab as a treatment for patients with refractory generalized MG, and this trial is expected to start in early 2014. Hematology Cold Agglutinin Disease (CAD): The company is aware that dosing is ongoing in an investigator-initiated Phase II study of eculizumab in patients for the treatment of CAD. CAD is a severe, ultra-rare complement-mediated autoimmune disease characterized by the presence of high concentrations of circulating complement-activating antibodies directed against red blood cells. Asfotase Alfa Hypophosphatasia (HPP): HPP is an ultra-rare, genetic, and life-threatening metabolic disease characterized by impaired phosphate and calcium regulation, leading to progressive damage to multiple vital organs, including destruction and deformity of bones, profound muscle weakness, seizures, impaired renal function, and respiratory failure. Asfotase alfa, a targeted enzyme replacement therapy in Phase II clinical trials for patients with HPP, is designed to directly address the morbidities and mortality of HPP by targeting alkaline phosphatase directly to the deficient tissue. cPMP (ALXN 1101) Molybdenum Cofactor Deficiency (MoCD) Disease Type A (MoCD Type A): MoCD Type A i


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