AZD 6244 filed with FDA for NF1 and plexiform neurofibromas and given priority review.- AstraZeneca and Merck Inc

AstraZeneca and Merck Inc announced that the FDA has accepted a New Drug Application (NDA) and granted Priority Review for AZD 6244 (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. A Prescription Drug User Fee Act (PDUFA) date is set for the second quarter of 2020.

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 at least 20% tumour volume reduction.

Comment: Selumetinib, a MEK 1/2 inhibitor 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.