Policy & Regulation
AstraZeneca and MSD announce FDA acceptance of NDA for selumetinib in neurofibromatosis type 1 and Priority Review
15 November 2019 -

AstraZeneca Plc (LON:AZN), a science-led biopharmaceutical company, and pharmaceutical company MSD Inc (Merck & Co Inc in US and Canada) (NYSE:MRK) announced on Thursday that the US Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) 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).

Reportedly, 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.

This regulatory submission was based on positive results from the National Cancer Institute (NCI) Cancer Therapy Evaluation Programme (CTEP)-sponsored SPRINT phase II Stratum 1 trial.

MEK 1/2 inhibitor selumetinib was granted 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 licence agreement. In July 2017, AstraZeneca and MSD announced a global strategic oncology collaboration to co-develop and co-commercialise Lynparza, the world's first PARP inhibitor, and potential new medicine selumetinib, a MEK inhibitor, for multiple cancer types.

Under this collaboration, the companies will develop Lynparza and selumetinib in combination with other potential new medicines and as monotherapies. Independently, the companies will develop Lynparza and selumetinib in combination with their respective PD-L1 and PD-1 medicines.

AstraZeneca is focussed on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of diseases in the three therapy areas of Oncology, Cardiovascular, Renal and Metabolism, and Respiratory.