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EC grants new indication for Soliris to treat refractory generalized myasthenia gravis

Published 22 August 2017

Alexion Pharmaceuticals announced that 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.1-4 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.5-7 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.8-10

Soliris is a first-in-class complement inhibitor that specifically and effectively inhibits the terminal part of the complement cascade.

“Our deep understanding of complement-mediated diseases enabled us to develop Soliris for the treatment of patients with refractory gMG,” said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. “We are grateful to the investigators and patients who participated in our clinical program, and we are excited about the opportunity to bring Soliris to patients who continue to suffer from this debilitating disease despite current therapies.”

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, 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.

Soliris® is a first-in-class complement inhibitor that works by inhibiting the terminal part of the complement cascade, a part of the immune system that, when activated in an uncontrolled manner, plays a role in serious ultra-rare disorders like paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS) and anti-acetylcholine receptor (AChR) antibody-positive refractory generalized myasthenia gravis (gMG).

Soliris is approved in the U.S., EU, Japan and other countries as the first and only treatment for patients with PNH and aHUS, and in the EU as the first and only treatment for refractory gMG in patients who are anti-AChR antibody-positive. Soliris is not indicated for the treatment of patients with Shiga-toxin E. coli-related hemolytic uremic syndrome (STEC-HUS).

Alexion and Soliris have received some of the pharmaceutical industry's highest honors for the medical innovation in complement inhibition: the Prix Galien USA (2008, Best Biotechnology Product) and France (2009, Rare Disease Treatment).



Source: Company Press Release