AstraZeneca, MSD's Koselugo Approved in US for NF1 Plexiform Neurofibromas
The US Food and Drug Administration (FDA) has approved Koselugo (selumetinib), developed by AstraZeneca and MSD, for children aged two and older with neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas (PN).
AstraZeneca and MSD have received US Food and Drug Administration (FDA) approval for Koselugo (selumetinib). The drug is indicated for paediatric patients aged two years and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN).
The approval follows positive results from the National Cancer Institute (NCI)-sponsored SPRINT trial. Koselugo is the first medicine approved globally for the treatment of NF1-related PN. NF1 is a rare genetic condition where PN can cause pain, motor dysfunction, and other clinical issues.
In the SPRINT study, 66% of paediatric patients treated with Koselugo achieved an objective response rate, defined as at least a 20% reduction in tumour volume. AstraZeneca and MSD are jointly developing and commercialising Koselugo, and marketing applications are also under review by the European Medicines Agency.
AstraZeneca received a Priority Review Voucher (PRV) which is intended to encourage the development of new medicines for rare paediatric diseases. The companies anticipate future sales and see the approval as a significant advancement for patients.