EU CHMP recommends Soliris (eculizumab) to include the treatment of refractory generalized myasthenia gravis (gMG) in patients who are anti-acetylcholine receptor (AChR) antibody-positive.-Alexion Pharma

Alexion Pharmaceuticals, Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion to extend the current therapeutic indication for Soliris (eculizumab) to include the treatment of refractory generalized myasthenia gravis (gMG) in patients who are anti-acetylcholine receptor (AChR) antibody-positive. The final decision from the European Commission (EC) is anticipated in the third quarter of 2017. If approved, Soliris will be the first treatment available in the European Union (EU) for patients with refractory gMG who are anti-AChR antibody-positive, and the first and only complement inhibitor approved for this disease.

Patients with refractory gMG have difficulties walking, talking, swallowing and breathing normally. Exacerbations and crises of their disease may require hospitalization and intensive care and may be life-threatening. Patients with refractory gMG who are anti-AChR antibody-positive represent an ultra-rare segment of patients with myasthenia gravis (MG). The CHMP based its opinion on comprehensive clinical data from the Phase III REGAIN study (MG-301) and its long-term extension study (MG-302). Soliris is approved in the United States ,EU, Japan and other countries for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), two ultra-rare, complement-mediated disorders. Alexion’s supplemental Biologics License Application (sBLA) in the U.S. and a supplemental new drug application in Japan for Soliris as a treatment for patients with anti-AChR antibody-positive refractory gMG have been accepted for review by the FDA and the Japanese Ministry of Health, Labour and Welfare (MHLW), respectively. Soliris has received Orphan Drug Designation (ODD) for the treatment of patients with MG in the U.S. and EU, and for the treatment of patients with refractory gMG in Japan.