AstraZeneca has announced that the National Medical Products Administration (NMPA) in China has granted approval for Koselugo (selumetinib) to treat symptomatic inoperable plexiform neurofibromas (PN) and neurofibromatosis type 1 (NF1) in paediatric patients aged three years and over.
Koselugo is a mitogen-activated protein kinase (MEK) inhibitor that works by blocking the enzymes MEK1 and MEK2 that are involved in stimulating cell growth.
It is claimed to be the first and only therapy to receive approval in the country to treat rare and progressive genetic conditions, which may result in the growth of benign tumours along nerves throughout the body.
The therapy has also received approval in countries and regions including the EU, Japan and the US to treat symptomatic, inoperable PNs and NF1 in paediatric patients.
AstraZeneca’s subsidiary Alexion CEO Marc Dunoyer said: “Koselugo offers hope for children whose quality of life and overall well-being is impacted by the growth of painful, debilitating tumours throughout the body.
“We are proud to bring forward a new, innovative treatment option to the NF1 community in China, delivering on Alexion’s commitment to reach more people living with rare diseases in China, and transform the lives of patients and caregivers across the globe.”
The regulatory approval was based on data obtained from the SPRINT Stratum 1 trial, backed by the National Cancer Institute’s (NCI) cancer therapy evaluation programme (CTEP).
The trial was designed to assess the objective response rate and impact on patient-reported and functional outcomes in NF1-related inoperable PNs paediatric patients treated with Koselugo (selumetinib) alone.
The findings showed that Koselugo reduced the inoperable tumour size in children.
Participants who received Koselugo twice daily orally had a 66% objective response rate (ORR).
Diarrhoea, vomiting, nausea and blood creatine phosphokinase increase were the most common adverse reactions observed in the SPRINT trial.