Soliris (eculizumab) receives marketing authorization in Japan for the treatment of patients with generalized myasthenia gravis (gMG).
Japan’s Ministry of Health, Labour and Welfare has approved Alexion Pharmaceuticals, Inc.’s Soliris (eculizumab) as a treatment for patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive and whose symptoms are difficult to control with high-dose intravenous immunoglobulin therapy or plasmapheresis.
Soliris is the first and only complement inhibitor approved in Japan as a treatment for these patients.
In the Phase 3 REGAIN study and its ongoing open-label extension study, Soliris demonstrated treatment benefits for patients with anti-AChR antibody-positive gMG who had previously failed immunosuppressive treatment and continued to suffer from significant unresolved disease symptoms, which can include difficulties seeing, walking, talking, swallowing, and breathing.1,2,3 These patients are at an increased risk of disease exacerbations and crises that may require hospitalization and intensive care and may be life-threatening.4,5
Chronic uncontrolled activation of the complement system, a part of the immune system, plays a major role in the debilitating symptoms and potentially life-threatening complications for patients with gMG who are anti-AChR antibody-positive.6,7,8 By selectively and effectively inhibiting the terminal complement cascade, Soliris targets a critical underlying cause of the disease.
Japan’s Ministry of Health, Labour and Welfare based its approval of this new indication of Soliris on comprehensive clinical data from the Phase 3, randomized, double-blind, placebo-controlled, multicenter REGAIN study (ECU-MG-301).
Soliris is also approved in the EU for the treatment of refractory gMG in adults who are anti-AChR antibody-positive, and in the U.S. for the treatment of adult patients with gMG who are anti-AchR antibody-positive.
__________________________________________________________
References:
1 Howard JF, Barohn RJ, Cutter GR, et al. A randomized, double-blind, placebo-controlled phase II study of eculizumab in patients with refractory generalized myasthenia gravis. Muscle Nerve. 2013;48(1):76-84.
2 National Institute of Neurological Disorders and Stroke. Myasthenia Gravis Fact Sheet. Publication date May 2017. https://www.ninds.nih.gov/disorders/myasthenia_gravis/detail_myasthenia_gravis.htm. Accessed October 12, 2017
3 Sathasivam S. Diagnosis and management of myasthenia gravis. Progress in Neurology and Psychiatry. January/February 2014.
4 Souayah N, Nasar A, Suri MF, et al. Trends in Outcomes and Hospitalization Charges among Mechanically Ventilated Patients with Myasthenia Gravis in the United States. Int J Biomed Sci. 2009;5(3):209-214.
5 Engel-Nitz N, et al. Clinical Burden of Refractory Generalized Myasthenia Gravis in the United States. Poster 146; ICNMD 2016.
6 Conti-Fine, et al. Myasthenia gravis: past, present, and future. J Clin Invest. 2006;116:2843-2354.
7 Tüzün E, Huda R, Christadoss P. Complement and cytokine based therapeutic strategies in myasthenia gravis. J Autoimmun. 2011;37(2):136-143.
(Source: Alexion Pharmaceuticals, Inc.)
Filed Under: Drug Discovery
