AstraZeneca and MSD’s Koselugo®▼(Selumetinib) has been granted Conditional Marketing Authorisation in Great Britain by the Medicines & Healthcare products Regulatory Agency (MHRA) as monotherapy for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in paediatric patients with neurofibromatosis type 1 (NF1) aged 3 years and above.
This is the first approval of a medicine for NF1 PN – a debilitating genetic condition – in the UK. Conditional marketing authorisation is based on positive results from the SPRINT Stratum 1 Phase II open-label, multicentre, single-arm trial sponsored by the National Institute of Health’s National Cancer Institute (NCI) Cancer Therapy Evaluation Program (CTEP). The trial evaluated the efficacy of selumetinib in 50 children and adolescents with NF1 and inoperable PN.
In the trial, selumetinib demonstrated an objective response rate (ORR) of 66%; 33 of 50 confirmed partial response (95% CI: 51.2-78.8) in paediatric patients with NF1 PN when treated with selumetinib as twice-daily oral monotherapy. ORR is defined as the percentage of patients with confirmed complete (disappearance of PN) or partial response (at least 20% reduction in tumour volume).8,9 Safety and efficacy data from the SPRINT Phase II trial with longer follow up will be provided as one of the conditions of the approval.
Prof. Gareth Evans, Manchester University NHS Foundation Trust, St Mary’s Hospital said: “Children living with neurofibromatosis type 1 have various unmet medical needs, limited treatment options and are often presented with significant quality of life challenges. The authorisation of selumetinib marks an important treatment advance for those living with the debilitating impacts of this disease.”
Arun Krishna, Head of Oncology, AstraZeneca U.K said: “We are delighted to receive conditional MHRA authorisation for selumetinib, the first approved medicine for neurofibromatosis type 1 in Great Britain. We are committed to helping people living with neurofibromatosis type 1 and believe that selumetinib has the potential to transform the management of plexiform neurofibromas. Our focus is now on securing access for NHS patients as quickly as possible.”