Alexion submits application to Japan’s MHLW to extend Soliris indication to treat gMG


Alexion Pharmaceuticals has submitted an application to extend the indication for Soliris (eculizumab) as a potential treatment for patients with refractory generalised myasthenia gravis (gMG).

Submitted to Japan’s Ministry of Labour and Welfare (MHLW), the application is supported by data from the Phase III Regain study.

Alexion Pharmaceuticals R&D executive vice-president and global head Martin Mackay said: “We look forward to working with the Japanese health authorities to bring this potentially life-transforming treatment to patients in Japan who are suffering and in urgent need of an effective therapy.

“If approved, Soliris could be a critical step toward improving the lives of patients with refractory gMG.”

Patients with AChR-positive refractory gMG represent an ultra-rare population and continue to face severe complications, including difficulty walking, talking, swallowing, and breathing normally, despite existing treatment options for gMG.

"If approved, Soliris could be a critical step toward improving the lives of patients with refractory gMG."

The company’s marketing applications in the US and EU to extend the indication for Soliris have been accepted by the US Food and Drug Administration (FDA) and validated by the European Medicines Agency (EMA).

Soliris has also received orphan drug designation (ODD) to treat patients with MG in the US and EU, and for treatment of patients with refractory gMG in Japan.

MG is a chronic, debilitating and progressive autoimmune neuromuscular disease where the complement system mediates a progressive, destructive inflammatory effect on the neuromuscular junction

Patients with refractory AChR-positive gMG experience severe morbidities and life-threatening complications.

Soliris has been developed from the laboratory through regulatory approval and commercialisation by Alexion and is approved in the US (2007), European Union (2007), Japan (2010) and other countries to treat patients with paroxysmal nocturnal haemoglobinuria (PNH) to reduce haemolysis.