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Then the manufacturer applies to the FDA for approval for to treat new indications with a new drug application (NDA). Sr. Director, Corporate Communications - At 48 weeks, 98% of patients treated with SOLIRIS were relapse free compared to 63% of patients receiving placebo - - SOLIRIS is the first and only FDA approved … Alexion will host a conference call/webcast that is scheduled for Monday, September 26, 2011 at 10:00 a.m., Eastern Time to discuss the FDA approval. A Phase II Study of Eculizumab in Patients With Atypical Hemolytic Uremic Syndrome Receiving Chronic Plasma Exchange/Infusion [poster].. The U.S. prescribing information for Soliris includes the following warnings and precautions: Life-threatening and fatal meningococcal infections have occurred in patients treated with Soliris. REGAIN clinical trial tested Soliris for MG. Profound weakness throughout the body often is accompanied by slurred speech, impaired swallowing and choking, double vision, upper and lower extremity weakness, disabling fatigue, shortness of breath due to respiratory muscle weakness, and episodes of respiratory failure. 3. The FDA’s approval decision date is Oct. 19. Under the Soliris REMS, prescribers must enroll in the program (5.2). Clinically meaningful improvements in several secondary measures were observed in patients treated with Soliris compared with those who received a placebo, including an improvement in the QMG score, a physician-administered test that assesses MG severity. MDA has invested in previous basic research studies investigating complement inhibition as a therapeutic strategy for MG and is currently funding studies aiming to develop improved complement inhibitor drugs for MG. The FDA approval of Soliris in aHUS is based on data from two prospective pivotal Phase 2 open-label clinical trials in adolescent and adult patients with aHUS, and a third retrospective study in children, adolescents, and adults with aHUS. Alexion is evaluating other potential indications for Soliris and is pursuing development of other innovative biotechnology product candidates in early stages of development. Generalized MG affects a subset of MG patients. Tsai HM. Treatment with Soliris will not cure generalized MG, but it may improve disease symptoms, the ability to carry out activities of daily living and quality of life. Earlier today, Alexion announced that the European Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending that the therapeutic indication for Soliris be extended to include the treatment of pediatric and adult patients with aHUS. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for meningococcal vaccination in patients with complement deficiencies. Hematologic normalization was observed in 13 of 17 Soliris-treated patients (76%). A statistically significant difference favoring Soliris was observed in the mean change from baseline to Week 26 in MG-ADL total scores (-4.2 points in the Soliris treated group compared with -2.3 points in the placebo-treated group). Hematologic normalization was achieved in 18 of 20 Soliris-treated patients (90%). SOLIRIS is used to treat: adults with a disease called neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody positive It is not known if SOLIRIS is safe and effective in children with NMOSD. Treatment effects were measured across four MG-specific assessment scales (MG-ADL, Quantitative Myasthenia Gravis [QMG], MG Composite [MGC] and MG Quality of Life 15 [MG-QoL 15]). By using our website, you agree to our use of cookies in accordance with our, Soliris® (eculizumab) Approved by FDA for All Patients with Atypical Hemolytic Uremic Syndrome (aHUS). Curr Opin Nephrol Hypertens 2010 May;19(3):242-7. Soliris also reduced the need for hospitalizations and the need for treatment of acute attacks with corticosteroids and plasma exchange. "This is the first time I have seen a therapy with such a dramatic benefit, including restored kidney function. (See Serious Meningococcal Infections (5.1) for additional guidance on the management of meningococcal infection.) In MG, antibodies whose job it is to target these toxic pathogens, instead inappropriately recruit the complement system and target the space across which nerve fibers transmit signals to muscle fibers, called the neuromuscular junction (NMJ). Alexion is the global leader in complement inhibition, and has developed and markets Soliris® (eculizumab) as a treatment for patients with PNH and aHUS, two debilitating, ultra-rare and life-threatening disorders caused by chronic uncontrolled complement activation. Soliris was first approved by the FDA in 2007. The phase 3 REGAIN study tested Soliris in people with generalized MG who had previously failed immunosuppressive treatment and continued to suffer from significant unresolved disease symptoms such as difficulties seeing, walking, talking, swallowing and breathing, and who also had an MG-ADL score of greater than or equal to 6. The Muscular Dystrophy Association (MDA) is a qualified 501(c)(3) tax-exempt organization. A drug like Soliris may launch with label approval to treat one condition. Bresin E, Daina E, Noris M, et al; International Registry of Recurrent and Familial HUS/TTP. In addition, a brief financial update will be provided. It is not known if SOLIRIS is safe and effective in children with gMG. Benz K, Amann K. Thrombotic microangiopathy: new insights. Genetics of HUS: the impact of MCP, CFH, and IF mutations on clinical presentation, response to treatment, and outcome. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early. Patients treated with Soliris also achieved statistically significant reduction in the TMA intervention rate, maintained or improved kidney function, and improved quality of life. 2,3 More than half of all patients with aHUS die, require kidney dialysis or have permanent kidney damage within 1 year of diagnosis.4 Patients with aHUS who receive a kidney transplant commonly experience subsequent systemic TMA, resulting in a 90% transplant failure rate.8, aHUS affects both children and adults. "Soliris directly targets uncontrolled complement activation, the underlying cause of the progressive organ failure and shortened life span of patients with aHUS, an extremely rare and devastating disease," said Larry Greenbaum, M.D., Ph.D., Director of Pediatric Nephrology at Emory University and Children's Healthcare of Atlanta. ", The FDA approval of Soliris in aHUS is based on data from two prospective pivotal Phase 2 open-label clinical trials in adolescent and adult patients with aHUS, and a third retrospective study in children, adolescents, and adults with aHUS. Soliris® (eculizumab) When requestingSoliris® (eculizumab), the individual requiring treatment must be diagnosed with an FDA-approved indicationand meet the specific coverage guidelines and applicable safety criteria for the covered indication. Enrollment in the Soliris REMS program and additional information are available by telephone: 1-888-SOLIRIS (1-888-765-4747). Soliris is the first in a new class of drugs to be approved for MG in the U.S. Soliris is not a cure, but it may lessen the symptoms experienced by people living with generalized MG. Soliris was tested in clinical trials in people who had previously failed immunosuppressive treatment and continued to suffer from significant unresolved disease symptoms such as difficulties seeing, walking, talking, swallowing and breathing. The Myasthenia Gravis-Specific Activities of Daily Living scale (MG-ADL) is a categorical scale that assesses the impact on daily function of eight signs or symptoms that are typically affected in generalized MG. Each item is assessed on a 4-point scale, where a score of 0 represents normal function and a score of 3 represents loss of ability to perform that function (total score 0-24). Participants who originally were in the placebo group of REGAIN also saw benefits from taking Soliris after 26 weeks of treatment. The inappropriate binding of antibodies to acetylcholine receptors activates the complement system, which leads to a localized destruction of the NMJ. Eculizumab was approved by the US Food and Drug Administration (FDA) in March 2007 for the treatment of PNH, Eculizumab has exclusivity rights until 2017, which protects it from competition until 2017.: 6 When the FDA approved it in September 2011 for the treatment of aHUS, it designated it as an orphan drug. Alexion's Soliris, which has is approved to treat the condition, reduced the risk of relapse by 94% over one-year of follow-up. Enrollment in the Soliris REMS program and additional information are available by telephone: 1-888-soliris (1-888-765-4747).". 8. 2006;108:1267-1279. Soliris is approved in the U.S., European Union, Japan and other countries as the first treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH), a debilitating, ultra-rare and life-threatening blood disorder, characterized by complement-mediated hemolysis (destruction of red blood cells). This website is intended only for residents of the United States. Soliris is the first in a new class of drugs to be approved for MG in the U.S. It was initially approved to treat paroxysmal nocturnal hemoglobinuria, a rare blood disorder. Be sure to check back at mda.org for updates as they become available. In patients with aHUS, the most frequently reported adverse events observed with Soliris treatment in clinical studies were hypertension, upper respiratory tract infection, and diarrhea. More information on Soliris is available at www.soliris.net. The ongoing prospective clinical trials are designed with the same endpoints as the completed trials. Forward-looking statements are subject to factors that may cause Alexion's results and plans to differ from those expected, including for example, decisions of regulatory authorities regarding marketing approval or material limitations on the marketing of Soliris for its current or potential new indications, and a variety of other risks set forth from time to time in Alexion's filings with the Securities and Exchange Commission, including but not limited to the risks discussed in Alexion's Quarterly Report on Form 10-Q for the period ended June 30, 2011, and in Alexion's other filings with the Securities and Exchange Commission. Soliris is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS). These are life-threatening genetic diseases that cause the breakdown of red blood cells resulting in various medical complications. FDA-approved Indications Soliris … 6. 2006;1:88-99. Patients had an ADAMTS13 activity level greater than 5%. Photos/Multimedia Gallery Available: http://www.businesswire.com/cgi-bin/mmg.cgi?eid=6870452&lang=en, Alexion Pharmaceuticals, Inc. Soliris is not indicated for the treatment of patients with Shiga toxin E coli-related hemolytic uremic syndrome (STEC-HUS).5 Alexion is currently evaluating the safety and efficacy of eculizumab for the treatment of patients with STEC-HUS. In patients with generalized MG who are anti-acetylcholine receptor antibody-positive, the most frequently reported adverse reaction observed with Soliris treatment in the placebo-controlled clinical study was musculoskeletal pain. 4. Eculizumab Efficacy and Safety in Patients With Atypical Hemolytic Uremic Syndrome (aHUS) Resistant to Plasma Exchange/Infusion [poster].