The FDA approval was based on positive data from the National Cancer Institute (NCI) Cancer Therapy Evaluation Programme (CTEP)-sponsored phase II SPRINT Stratum 1 study organised by the NCI’s Center for Cancer Research, Paediatric Oncology Branch.
According to AstraZeneca, the results demonstrated an overall response rate (ORR) of 66% in paediatric patients with NF1 PN when treated with Koselugo as a twice-daily oral monotherapy.
The SPRINT phase I/II trial has been framed to assess the objective response rate and impact on patient-reported and functional outcomes in paediatric patients with NF1-related inoperable PNs treated with Koselugo monotherapy.
AstraZeneca oncology business unit executive vice president Dave Fredrickson said: “For the first time, patients and families impacted by this incurable genetic condition have an approved medicine to treat the resulting plexiform neurofibromas.”
Koselugo is an inhibitor of mitogen-activated protein kinase kinases 1 and 2 (MEK1/2), which are upstream regulators of the extracellular signal-related kinase (ERK) pathway.
Koselugo secured FDA breakthrough therapy designation in April 2019, while rare paediatric disease designation in December 2019 and orphan drug designation in February 2018. It also secured EU orphan designation in August 2018 and Swissmedic orphan drug status in December 2018 to treat paediatric patients with NF1 PN.
AstraZeneca and Merck, which are jointly developing and commercialising Koselugo across the globe, submitted a marketing authorisation application in NF1 PN to the European Medicines Agency (EMA) in the first quarter of this year.
AstraZeneca will book all monotherapy product sales of Koselugo in line with the existing collaboration agreement between both firms. Merck will secure half of the gross profits related to the product.
In July 2017, AstraZeneca and Merck collaborated to co-develop and co-commercialise Lynparza PARP inhibitor and Koselugo MEK inhibitor for multiple cancer types.
MSD Research Laboratories chief medical officer, global clinical development head and senior vice president Roy Baynes said: “Previously there were no medicines approved for this disease. This approval has the potential to change how symptomatic, inoperable NF1 plexiform neurofibromas are treated and provides new hope for these patients.”
In January 2020, AstraZeneca secured orphan drug designation (ODD) from the FDA for Imfinzi (durvalumab) and tremelimumab to treat liver cancer called hepatocellular carcinoma (HCC).