Priority review for selumetinib to treat NF1

January 6, 2020 0 By FM

AstraZeneca and MSD announced that the US FDA has granted Priority Review for selumetinib as a potential new medicine for paediatric patients aged three years and older with neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas (Pns).

This is the first acceptance of a regulatory submission for an oral monotherapy for the treatment of NF1, a rare and incurable genetic condition.

The regulatory submission was based on positive results from the National Cancer Institute (NCI) Cancer Therapy Evaluation Program (CTEP)-sponsored SPRINT phase II Stratum 1 trial. An Objective Response Rate (ORR) was achieved in 66% of paediatric patients with NF1 and symptomatic, inoperable PNs (n=33/50 patients) when treated with selumetinib as a twice-daily oral monotherapy. ORR was defined as the percentage of patients with a confirmed complete or partial response of = 20% tumour volume reduction.

Selumetinib is a MEK 1/2 inhibitor. It is designed to inhibit the MEK enzyme in the RAS/MAPK pathway, a cell-signalling pathway, associated with cancer cell growth and proliferation in a number of different tumour types.

Selumetinib was granted US FDA Breakthrough Therapy Designation in April 2019, Orphan Drug Designation in February 2018, EU Orphan Designation in August 2018 and Swissmedic Orphan Drug Status in December 2018.