Soliris (eculizumab) has been approved in China for the treatment of adult patients with refractory generalised myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive. Soliris is the first and only complement inhibitor approved for the treatment of gMG in China.
The approval by the National Medical Products Administration (NMPA) in China was based on comprehensive results from the Phase III REGAIN trial. In the trial, Soliris demonstrated clinical benefit for patients with anti-AChR antibody-positive gMG who had previously failed immunosuppressive treatment and continued to suffer from significant unresolved disease symptoms. These patients are at an increased risk of disease exacerbations and crises that may require hospitalisation and intensive care and may be life-threatening.
Additionally, improvements demonstrated during the initial six-month duration of the REGAIN trial were sustained over a treatment period of more than 130 weeks in the long-term open-label extension trial.
gMG is a rare, debilitating, chronic, autoimmune neuromuscular disease that leads to a loss of muscle function and severe weakness.
Yan Chuanzhu, MD, Deputy President and Director of the Department of Neurology at Qilu Hospital of Shandong University, Jinan, China, said: “For some gMG patients, standard treatments such as immunosuppressants may not sufficiently control the disease, leaving patients at risk of serious complications.
“The approval of Soliris is an important milestone for the gMG community in China, offering a targeted treatment option with significant clinical data and extensive real-world evidence shown to improve the ability to perform activities of daily living and support quality of life.”
Marc Dunoyer, Chief Executive Officer, Alexion, said: “Symptoms of gMG, including difficulties seeing, walking, talking, swallowing and breathing, can have a debilitating impact on daily life for patients and their families, representing a critical need for therapeutic advances.
“We are proud to offer Soliris in China, a first-in-class C5 complement inhibitor and globally established treatment for gMG. We remain committed to expanding access to innovative therapies for rare disease patients in China and around the world.”
The safety and tolerability profile of Soliris were consistent throughout the primary treatment period and open-label extension. The most common adverse events in the primary treatment period were headache and upper respiratory tract infection.
Soliris recently became available in China for the treatment of paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS) in adults and children. Soliris is also approved for multiple indications in many countries around the world.
AstraZeneca established a rare disease business unit in China in September 2021. In the future, the company aims to continue introducing more innovative medicines in China, targeting the complement system and beyond, for the treatment of rare diseases including PNH, aHUS, gMG, neuromyelitis optica spectrum disorder (NMOSD), hypophosphatasia, immunoglobulin A nephropathy, lupus nephritis and amyloidosis.