The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended conditional marketing authorisation for AstraZeneca and Merck’s selumetinib to treat paediatric patients aged three years and older with neurofibromatosis type 1 (NF1), symptomatic and inoperable plexiform neurofibromas (PN).
Merck is known as MSD outside the US and Canada.
Selumetinib is a mitogen-activated protein kinase kinases 1 and 2 (MEK1/2) inhibitor, which received Breakthrough Therapy Designation from the US Food and Drug Administration (FDA) in April 2019 and the US Orphan Drug Designation in February 2018.
It has also secured orphan drug designations in the European Union (EU), Japan, Russia, Switzerland, South Korea, Taiwan and Australia.
The CHMP’s recommendation for EU approval was based on data from the National Cancer Institute (NCI) Cancer Therapy Evaluation Program (CTEP)-sponsored SPRINT Stratum 1 phase II trial, which showed selumetinib reduced tumour volume in children.
Oncology Business Unit executive vice president Dave Fredrickson said: “This recommendation means patients in the EU are one step closer to receiving the only approved medicine for neurofibromatosis type 1 and the only treatment outside of surgery, which is not an option for many patients. Children living with this rare genetic condition are in great need of novel treatment options to help address the impact of this disease.”
The trial has shown increase in objective response rate (ORR) by 66% in paediatric patients suffering with NF1 PN when treated with selumetinib as twice-daily oral monotherapy.
ORR is the patient’s percentage with complete or partial response of at least 20% reduction in the tumour volume.2
MSD Research Laboratories chief medical officer and Global Clinical Development head and senior vice president Roy Baynes said: “In the SPRINT trial, selumetinib was shown to reduce the size of these inoperable tumours, a meaningful clinical advance for children living with this debilitating disease.”