US FDA accepts regulatory submission for selumetinib in neurofibromatosis type 1 and grants priority review

AstraZeneca

14 November 2019 - AstraZeneca and MSD’s selumetinib would become the first medicine indicated for the treatment of paediatric patients with NF1 plexiform neurofibromas if approved.

AstraZeneca and MSD today announced that the US FDA has accepted a new drug application and 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. A Prescription Drug User Fee Act 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 ≥ 20% tumour volume reduction.

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. AstraZeneca and MSD are jointly developing and commercialising selumetinib globally under a license agreement.

Read AstraZeneca press release

Michael Wonder

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Michael Wonder

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Medicine , US , Priority review , Dossier