Koselugo (selumetinib) has been approved in China for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in paediatric patients with neurofibromatosis type 1 (NF1) aged three years and above.

“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,” says Marc Dunoyer, Chief Executive Officer, Alexion.

The SPRINT Stratum 1 trial

The approval by the National Medical Products Administration (NMPA) in China was based on positive results from the SPRINT Stratum 1 trial sponsored by the National Institutes of Health’s National Cancer Institute (NCI) Cancer Therapy Evaluation Program (CTEP). The trial showed Koselugo, an oral treatment option, reduced the size of inoperable tumours in children.

Results from the SPRINT Stratum 1 Phase II trial showed that Koselugo demonstrated an objective response rate (ORR) of 66% (33 of 50 patients, confirmed partial responses) in paediatric patients with PNs in NF1 when treated with Koselugo as twice-daily oral monotherapy. ORR is defined as the percentage of patients with confirmed complete (disappearance of PNs) or partial response (at least 20% reduction in tumour volume). The most common adverse reactions in the SPRINT trial were vomiting, blood creatine phosphokinase increase, diarrhoea and nausea.

In addition to China, Koselugo is also approved in the US, EU, Japan and several other countries for the treatment of paediatric patients with NF1 and symptomatic, inoperable PNs.

Photo of Marc Dunoyer: AstraZeneca