"The orphan drug designation for eculizumab for NMO highlights the significant need for an effective and innovative treatment option for patients in
The MHLW, based on the opinion of the
Alexion is enrolling patients in a multinational, placebo-controlled registration trial of eculizumab in patients with relapsing NMO, known as the PREVENT (Prevention of Relapses and EValuation of Eculizumab in NMO Treatment) study. More information on this trial is available at www.clinicaltrials.gov under the identifier NCT01892345.
Soliris is a first-in-class terminal complement inhibitor and is currently approved in
About Neuromyelitis Optica
In patients with neuromyelitis optica (NMO), binding of NMO-IgG antibody to astrocytes results in uncontrolled complement activation and destruction of myelin-producing cells, leading to severe damage to the central nervous system and predominantly impacting the spinal cord and optic nerve.1-3 The disease is characterized by severe weakness, paralysis, respiratory failure, loss of bowel and bladder function, blindness and premature death.4-6 Patients with NMO have a life-long exposure to the uncontrolled terminal complement activation due to chronic autoimmune attack, and most patients experience an unpredictable, relapsing course of disease with cumulative disability, as each attack adds to the neurologic disability.5,7,8 Fifty percent of relapsing NMO patients have been reported to sustain permanent severe disability, including paralysis and blindness, within five years of disease onset.9 Most NMO-related deaths result from respiratory complications from NMO attacks.9,10 The disease primarily affects women, with a female to male ratio as high as a 9:1.11 Currently, there are no approved treatments for NMO.
About Soliris® (eculizumab)
Soliris is a first-in-class terminal complement inhibitor developed from the laboratory through regulatory approval and commercialization by Alexion. Soliris is approved in the U.S. (2007),
More information including the full U.S. prescribing information on Soliris is available at www.soliris.net.
Important Safety Information
The U.S. product label for Soliris includes a boxed warning: "Life-threatening and fatal meningococcal infections have occurred in patients treated with Soliris. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early [see Warnings and Precautions (5.1)]. Comply with the most current
In patients with PNH, the most frequently reported adverse events observed with Soliris treatment in clinical studies were headache, nasopharyngitis (runny nose), back pain and nausea. Soliris treatment of patients with PNH should not alter anticoagulant management because the effect of withdrawal of anticoagulant therapy during Soliris treatment has not been established. In patients with aHUS, the most frequently reported adverse events observed with Soliris treatment in clinical studies were headache, diarrhea, hypertension, upper respiratory infection, abdominal pain, vomiting, nasopharyngitis, anemia, cough, peripheral edema, nausea, urinary tract infections, pyrexia. Soliris is not indicated for the treatment of patients with Shiga-toxin E. coli-related hemolytic uremic syndrome (STEC-HUS). Please see full prescribing information for Soliris, including BOXED WARNING regarding risk of serious meningococcal infection.
About Alexion
Alexion is a biopharmaceutical company focused on serving patients with severe and rare disorders through the innovation, development and commercialization of life-transforming therapeutic products. 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 is currently approved in nearly 50 countries for the treatment of PNH, and in nearly 40 countries for the treatment of aHUS. Alexion is evaluating other potential indications for Soliris in additional severe and ultra-rare disorders beyond PNH and aHUS, and is developing other highly innovative biotechnology product candidates across multiple therapeutic areas. This press release and further information about Alexion can be found at www.alexionpharma.com.
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This news release contains forward-looking statements, including statements related to potential medical benefits of Soliris® (eculizumab) for the treatment of neuromyelitis optica (NMO). 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 NMO, delays in arranging satisfactory manufacturing capabilities, the possibility that results of clinical trials are not predictive of safety and efficacy results of Soliris for NMO in broader or different patient populations, decisions of regulatory authorities to require additional testing, the risk that estimates regarding the number of patients with NMO and observations regarding the natural history of patients with NMO are inaccurate, and a variety of other risks set forth from time to time in Alexion's filings with the
References
1. Jarius S, Wildemann B. AQP4 antibodies in neuromyelitis optica: diagnostic and pathogenetic relevance. Nat Rev Neuro. 2010;6:383-92.
2. Hinson SR, Romero MF, Popescu BFG, et al. Molecular outcomes of neuromyelitis optica (NMO)-IgG binding to aquaporin-4 in astrocytes. Proc Nat Acad Sci 2012;109(4):1245-50.
3. Hinson SR, Pittock SJ, Lucchinetti CF, et al. Pathogenic potential of IgG binding to water channel extracellular domain in neuromyelitis optica. Neurology 2007;69:2221-31.
4. Wingerchuk DM, Lennon VA, Lucchinetti CF, Pittock SJ, Weinshenker BG. The spectrum of neuromyelitis optica. Lancet Neuro. 2007;6(9):805-15.
5. Wingerchuk DM. Diagnosis and treatment of neuromyelitis optica. Neurologis. 2007;13(1):2-11.
6. Wingerchuk DM, Weinshenker BG. Neuromyelitis optica. Curr Treat Options Neurol 2008;10(1):55-66.
7. Tuzun E, Kurtuncu M, Turkoglu R, et al. Enhanced complement consumption in neuromyelitis optica and Behcet's disease patients. J Neuroimmunol 2011;233(1-2):211-5.
8. Kuroda H, Fujihara K, Takano R, et al. Increase of complement fragment C5a in cerebrospinal fluid during exacerbation of neuromyelitis optica. J Neuroimmunol 2013;254(1-2):178-82.
9. Wingerchuk DM, Hogancamp WF,O'Brien PC, Weinshenker BG. The clinical course of neuromyelitis optica (Devic's syndrome). Neurology 1999;53(5):1107-14.
10. Kitley J, Leite MI, Nakashima I, et al. Prognostic factors and disease course in aquaporin-4 antibody-positive patients with neuromyelitis optica spectrum disorder from the
11. Wingerchuk DM. Neuromyelitis optica. Int MS J 2006;13(2):42-50.
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