d in this Phase III registration trial study of eculizumab in patients at elevated risk for DGF following kidney transplant. Eculizumab has been granted orphan drug designation for DGF by the FDA, and in 2014, the EC granted orphan drug designation to eculizumab for the prevention of DGF after solid organ transplantation. In August 2014, the company announced the initiation of dosing in a single, multinational, placebo-controlled DGF registration trial based on positive discussions with regulators in the U.S. and EU. 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. Enrollment in a multi-national, multi-site controlled clinical trial of eculizumab in presensitized kidney transplant patients at elevated risk for AMR who received kidneys from deceased organ donors was completed in March 2013. The study was re-opened in October 2013 to enroll additional patients at the request of participating investigators. Enrollment and dosing in this expanded trial has been completed and patient follow-up in the trial is continuing. In September 2013, researchers presented positive preliminary data from the eculizumab deceased-donor AMR kidney transplant study at the European Society of Organ Transplant in Vienna, Austria. Enrollment in a multi-national, multi-site randomized controlled clinical trial of eculizumab in presensitized kidney transplant patients at elevated risk for AMR who received kidneys from living donors has been completed and patient follow-up in the trial is ongoing. In January 2015, the company reported results from a randomized, open-label, multicenter Phase II clinical trial to determine the safety and efficacy of eculizumab in the prevention of AMR in living-donor kidney transplant recipients requiring desensitization. The company is developing plans to commence a clinical trial with eculizumab as a treatment for patients with AMR. In April 2014, the EC granted orphan drug designation to eculizumab for the prevention of graft rejection following solid organ transplantation. Neurology Neuromyelitis Optica (NMO): NMO is a severe and ultra-rare autoimmune disease of the central nervous system that primarily affects the optic nerves and spinal cord. In an investigator-initiated Phase II clinical trial of eculizumab in severe and relapsing NMO, eculizumab reduced the median number of NMO attacks at 12 months with a high degree of statistical significance. In 2014, the company commenced a Phase III pivotal trial to evaluate eculizumab as treatment for patients with relapsing NMO. The FDA, EC, and MHLW have each granted orphan designation for eculizumab as a treatment for patients with NMO. Myasthenia Gravis (MG): MG is an ultra-rare autoimmune syndrome characterized by complement activation leading to the failure of neuromuscular transmission. Data from a Phase II trial evaluating the safety and efficacy of eculizumab in patients with refractory generalized MG indicated improvement in clinical measures. In 2014, the company commenced a Phase III pivotal trial to evaluate eculizumab as a treatment for patients with refractory generalized MG. In addition, the FDA, EC, and MHLW have granted orphan drug designation for eculizumab as a treatment for patients with MG. 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. In 2013, asfotase alfa received Breakthrough Therapy Designation from the FDA. In September 2014, the MHLW granted orphan drug designation to asfotase alfa for the treatment of patients with HPP.
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Cheshire, CT 06410
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