Alexion Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development and commercialization of life-transforming therapeutic products. In the company’s complement franchise, Soliris is the therapeutic approved for patients with either paroxysmal nocturnal hemoglobinuria (PNH); or atypical hemolytic uremic syndrome (aHUS). PNH and aHUS are two severe and ultra-rare disorders resulting from chronic uncontrolled activation of the complement component of the immune system. The company is developing Soliris (eculizumab) for the treatment of PNH Registry that is in Phase IV development stage. It is also developing Soliris (eculizumab) for the treatment of aHUS Registry that is Phase IV development stage. In its metabolic franchise, the company markets Strensiq for the treatment of patients with hypophosphatasia (HPP); and Kanuma for the treatment of patients with lysosomal acid lipase (LAL) deficiency (LAL-D). HPP is a genetic ultra-rare disease characterized by defective bone mineralization that can lead to deformity of bones and other skeletal abnormalities. LAL-D is a serious, life threatening ultra-rare disease in which genetic mutations result in decreased activity of the LAL enzyme leading to marked accumulation of lipids in vital organs, blood vessels and other tissues. The company is also evaluating additional potential indications for eculizumab in other severe and devastating diseases in which uncontrolled complement activation is the underlying mechanism; and is progressing in various stages of development with additional product candidates as potential treatments for patients with severe and life-threatening rare disorders. Soliris (eculizumab) Soliris is designed to inhibit a specific aspect of the complement component of the immune system and thereby treat inflammation associated with chronic disorders in various therapeutic areas, including hematology, nephrology, transplant rejection and neurology. Soliris is a humanized monoclonal antibody that blocks terminal complement activity at the doses prescribed. Soliris is a humanized monoclonal antibody that blocks terminal complement activity at the doses prescribed. The initial indication for which the company received approval for Soliris is PNH. The company works with researchers to expand the base of knowledge in PNH and the utility of Soliris to treat patients with PNH. Soliris is approved for the treatment of PNH in the United States, Europe, Japan and in various other territories. Soliris is approved for the treatment of pediatric and adult patients with aHUS in the United States, Europe and Japan. Strensiq, a targeted enzyme replacement therapy, is the approved therapy for patients with HPP, and is designed to directly address underlying causes of HPP. The company is developing Strensiq (asfotase alfa) for the treatment of HPP registry and is in Phase IV development stage. Kanuma (sebelipase alfa) LAL-D LAL-D is a serious, life-threatening ultra-rare disease associated with premature mortality and significant morbidity. Kanuma, a recombinant form of the human LAL enzyme, is the enzyme-replacement therapy that is approved for the treatment for patients with LAL-D. In 2015, the company announced that the U.S. Food and Drug Administration (FDA) approved Kanuma for the treatment of patients with LAL-D. The company is developing Kanuma (sebelipase alfa) for the treatment of LAL-D registry and is in Phase IV development stage. Soliris (eculizumab) Neurology The company is developing Soliris (eculizumab) for the treatment of myasthenia gravis (MG) and is in Phase III development stage. The FDA, the European Commission (EC), and Japan’s Ministry of Health Labour and Welfare (MHLW) granted orphan drug designation for eculizumab as a treatment for patients with MG. The company is developing Soliris (eculizumab) for the treatment of neuromyelitis optica spectrum disorder (NMOSD) and is in Phase III development stage. The FDA, EC, and MHLW have each granted orphan designation for eculizumab as a treatment for patients with NMOSD. Transplant The company is developing Soliris (eculizumab) for the treatm
alexion pharmaceuticals inc
100 College Street
New Haven, CT 06510
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