AstraZeneca (AZ) has received approval from China's National Medical Products Administration (NMPA) for a new paediatric indication for its complement C5 inhibitor, Soliris (eculizumab). The therapy is now approved for the treatment of refractory generalised myasthenia gravis (gMG) in children aged 6 and above who are anti-acetylcholine receptor (AChR) antibody-positive. This marks the first and only targeted therapy approved for this specific paediatric population in China.

The approval is based on positive results from the Phase III ECU-MG-303 trial, which demonstrated a statistically significant and clinically meaningful improvement in a key disease severity score after 26 weeks of treatment. Eculizumab, initially developed by Alexion (acquired by AZ in 2020), was first approved in China in 2018 for other rare diseases. It works by selectively inhibiting the terminal complement pathway. The therapy is already approved for adult gMG patients in China and for certain paediatric and adult gMG patients in multiple other regions, including EU, Japan, and the US.

According to PharmCube's NextBiopharm® database, eculizumab is approved for four diseases in China and is in early/middle stages of development in three other indications. Click here to request a free trial for NextBiopharm®.