For the treatment of adult patients with refractory generalized myasthenia gravis (gMG) and anti-acetylcholine receptor (AChR) antibody positivity, Soliris (eculizumab) has received approval in China. The only complement inhibitor authorized for the treatment of gMG in China is Soliris.
Comprehensive findings from the Phase III REGAIN study served as the foundation for the clearance by the National Medical Products Administration (NMPA) in China. In the trial, Soliris showed therapeutic benefit for individuals with gMG and anti-AChR antibody positivity who had previously had unsuccessful immunosuppressive therapy and who still had severe unresolved illness symptoms. These individuals are more likely to experience illness crises and exacerbations that might necessitate hospitalization, intensive care, and even be fatal.
Additionally, in the long-term open-label extension experiment, the gains that were shown during the first six-month phase of the REGAIN trial persisted beyond a treatment period of more than 130 weeks.
A rare, crippling, chronic autoimmune neuromuscular illness called gMG causes significant paralysis and muscle atrophy.
“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.”
– Yan Chuanzhu, MD, Deputy President and Director of the Department of Neurology at Qilu Hospital of Shandong University, Jinan, China
“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.”
– Marc Dunoyer, Chief Executive Officer, Alexion
Throughout the initial treatment phase and open-label extension, Soliris’ safety and tolerability profile remained constant. Headache and upper respiratory tract infection were the most frequent side effects during the initial therapy period.
For the treatment of both adults and children with PNH and atypical haemolytic uraemic syndrome (aHUS), Soliris has just been accessible in China. Additionally, Soliris is authorized in several nations across the world for a variety of indications.
In September 2021, AstraZeneca opened a commercial division for rare diseases in China. To treat rare diseases like PNH, aHUS, gMG, neuromyelitis Optica spectrum disorder (NMOSD), hypophosphatasia, immunoglobulin A nephropathy, lupus nephritis, and amyloidosis, the company plans to continue introducing more cutting-edge medications in China that target the complement system and beyond.