The NMPA approval was based on the results of Phase 3 REGAIN trial in which Soliris showed clinical benefit for patients with anti-AChR antibody-positive gMG
Soliris approved in China. (Credit: AstraZeneca)
AstraZeneca has announced that Soliris (eculizumab) has been approved by China’s National Medical Products Administration (NMPA) to treat adult patients with refractory generalised myasthenia gravis (gMG).
Manufactured by Alexion, a rare disease group in AstraZeneca, Soliris has been approved in China for patients who are anti-acetylcholine receptor (AChR) antibody-positive.
Soliris is a C5 complement inhibitor that inhibits the C5 protein in the terminal complement cascade. It is said to be the first and only complement inhibitor approved for gMG in China.
The NMPA approval was based on the results of the Phase 3 REGAIN trial which enrolled 125 patients with a confirmed diagnosis of refractory gMG with positive serologic test for antibodies against AChR.
In the trial, Soliris showed clinical benefit for patients with anti-AChR antibody-positive gMG who had previously failed immunosuppressive therapy and continued with major unresolved disease symptoms.
In addition, the improvements found during the initial six-month duration of the late-stage trial were sustained over a treatment period of more than 130 weeks in the long-term open-label extension trial.
US-based AstraZeneca said that the safety and tolerability profile of the C5 complement inhibitor remained constant throughout the initial treatment phase and open-label extension.
Alexion CEO Marc Dunoyer 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.”
REGAIN Phase 3 trial is a randomised, double-blind, placebo-controlled, multicentre 26-week trial in which the patients were randomised 1:1 to receive Soliris or a placebo for 26 weeks.
The primary efficacy endpoint was the change from baseline in MG-ADL total score at week 26. The three secondary endpoints included changes from baseline in Quantitative Myasthenia Gravis (QMG), Myasthenia Gravis Composite score and Myasthenia Gravis Quality of Life 15-item scale.