Selumetinib for paediatric patients with NF1 PN

May 9, 2020 0 By FM

AstraZeneca and MSD announced that the US FDA has approved selumetinib (Koselugo) for the treatment of paediatric patients two years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN).

The approval by the FDA was based on positive results from the National Cancer Institute (NCI) Cancer Therapy Evaluation Program (CTEP)-sponsored phase II SPRINT Stratum 1 trial coordinated by the NCI’s Center for Cancer Research, Paediatric Oncology Branch. 

This is the first regulatory approval anywhere in the world of a medicine for the treatment of NF1 PN.

Results showed an overall response rate (ORR) of 66% (33 of 50 patients, confirmed partial response) in paediatric patients with NF1 PN when treated with selumetinib as a twice-daily oral monotherapy. ORR is defined as the percentage of patients with confirmed complete or partial response of at least 20% reduction in tumour volume.

Selumetinib is inhibitor of mitogen-activated protein kinase kinases 1 and 2 (MEK1/2). MEK1/2 proteins are upstream regulators of the extracellular signal-related kinase (ERK) pathway. Both MEK and ERK are critical components of the RAS-regulated RAF-MEK-ERK pathway, which is often activated in different types of cancers.