AstraZeneca's Eculizumab has been approved in China for treating refractory generalized myasthenia gravis (gMG) in children aged 6+ who are AChR antibody-positive

On January 26, AstraZeneca announced that China's NMPA has officially approved the expanded indication of Soliris® (Eculizumab Injection) for the treatment of pediatric patients aged 6 years and older with refractory generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive. According to the press release, this marks the first and only targeted therapy approved in China for pediatric patients with this condition.

Source:AstraZeneca's Official Weibo account

gMG is a rare, debilitating, chronic autoimmune neuromuscular disorder that can lead to muscle function loss and severe debilitation.

Eculizumab is the world's first C5 complement inhibitor, functioning through selective inhibition of terminal complement C5 activation. The complement system is a crucial component of the human immune system, but when complement is activated in an uncontrolled manner, it may cause the body to attack its own healthy cells.

In 2023, eculizumab was approved in China for the treatment of adult patients with anti-acetylcholine receptor antibody-positive refractory generalized myasthenia gravis.

This recent approval is based on positive results from the Phase III clinical trial (ECU-MG-303) conducted in pediatric patients with refractory gMG. In this study, eculizumab demonstrated a highly statistically significant and clinically meaningful improvement in the Quantitative Myasthenia Gravis (QMG) total score at Week 26 compared to baseline (-5.8 [95% CI: -8.4, -3.13]; p=0.0004).

The efficacy and safety of eculizumab in pediatric patients aged 6 years and older with refractory gMG are consistent with its performance in clinical trials involving adult patients with refractory gMG.

Currently, eculizumab has been approved for multiple indications globally, including:

Approval in the United States, the European Union, Japan, China, and other countries for the treatment of specific pediatric and adult patients with paroxysmal nocturnal hemoglobinuria (PNH).

Approval in the United States, the European Union, Japan, China, and other countries for the treatment of specific pediatric and adult patients with atypical hemolytic uremic syndrome (aHUS).

Approval in the United States, the European Union, Japan, China, and other countries for the treatment of specific adult patients with generalized myasthenia gravis (gMG), as well as approval in the United States, the European Union, Japan, China, and other countries for the treatment of specific pediatric patients with gMG.

Additionally, eculizumab has been approved in the United States, the European Union, Japan, China, and other countries for the treatment of specific adult patients with neuromyelitis optica spectrum disorder (NMOSD).

Eculizumab is not indicated for the treatment of patients with Shiga toxin-producing E. coli-related hemolytic uremic syndrome (STEC-HUS).