The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended AstraZeneca and Merck’s (MSD) selumetinib for conditional marketing authorisation in the EU for treating children aged three years and above with neurofibromatosis type 1 (NF1) and plexiform neurofibromas (PN).
A mitogen-activated protein kinase kinases 1 and 2 (MEK1/2) inhibitor, selumetinib is approved in the US for treating paediatric patients with NF1 and symptomatic, inoperable PN.
The CHMP recommendation is based on data from the SPRINT Stratum 1 Phase II trial sponsored by the National Cancer Institute (NCI) Cancer Therapy Evaluation Program (CTEP).
According to results from this trial, selumetinib showed an objective response rate (ORR) of 66% in paediatric subjects with NF1 PN who received it as oral monotherapy two times a day.
As a condition of the recommendation for approval, safety and efficacy data from the trial with longer follow up will be submitted to the CHMP by the company.
AstraZeneca 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 company noted that additional regulatory submissions are progressing.
Furthermore, clinical trials of selumetinib in adult patients with NF1 PN and in an alternative age-appropriate formulation for paediatric patients are slated to start this year.
AstraZeneca and Merck had entered a global strategic oncology collaboration in 2017 to co-develop and co-commercialise PARP inhibitor, Lynparza and selumetinib for various cancer types.
Merck Research Laboratories global clinical development senior vice-president, head and chief medical officer 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.”