EU Commission Grants New Indication for Soliris for Refractory gMG

Alexion Pharmaceuticals announced the European Commission (EC) approved the extension of the indication for Soliris (eculizumab) to include the treatment of refractory generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) antibody-positive. Soliris is the first and only complement-based therapy approved in the European Union (EU) for this ultra-rare subset of patients. Patients with refractory gMG can have difficulties walking, talking, swallowing and breathing normally despite therapies currently used for MG. Exacerbations and crises of their disease may require hospitalization and intensive care and may be life-threatening. Soliris will be launched for this new indication initially in Germany, and Alexion is evaluating launches in additional EU countries.

"Patients with refractory gMG have exhausted multiple therapies and continue to suffer from severe symptoms and complications that markedly impact their daily lives," said Renato Mantegazza, MD, from the Department of Neuroimmunology and Neuromuscular Diseases, at the Istituto Neurologico Carlo Besta in Milan, Italy, and an investigator in the Phase 3 REGAIN study. "There is an urgent need for therapy for these patients, and it’s exciting to have a product such as Soliris available that has demonstrated in clinical studies that it improves patients’ symptoms and their ability to undertake daily activities."

Chronic uncontrolled activation of the complement cascade, a part of the immune system, can play a major role in the debilitating symptoms and potentially life-threatening complications of refractory gMG. Soliris is a first-in-class complement inhibitor that specifically and effectively inhibits the terminal part of the complement cascade.

The EC based its approval of the extended indication for Soliris on comprehensive clinical data from the Phase 3 REGAIN study (MG-301) and its long-term open-label extension study (MG-302).

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 U.S. Food and Drug Administration (FDA) and the Japanese Ministry of Health, Labor 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.