FDA grants priority review to AstraZeneca, Merck & Co.'s filing for selumetinib in neurofibromatosis type 1

AstraZeneca and Merck & Co. said Thursday that the FDA granted priority review to a marketing application seeking clearance of selumetinib for paediatric patients aged three years and older with neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas (PNs). The agency is scheduled to make a decision on the filing during the second quarter of 2020.

The companies noted that if approved, the MEK 1/2 inhibitor would become the first medicine indicated for the treatment of paediatric patients with NF1 plexiform neurofibromas.

The filing is backed by data from the Phase I/II SPRINT trial, which enrolled 50 patients aged two to 18 years old with NF1, inoperable PN and one or more PN-related morbidities, with disfigurement, motor dysfunction and pain being the most common in the study population. The trial assessed objective response rate (ORR), defined as the percentage of patients with a confirmed complete or partial response of at least 20% tumour volume reduction. Results demonstrated that 66% of patients achieved an ORR when treated with selumetinib as a twice-daily oral monotherapy.

Selumetinib, also known as AZD6244, was originally developed by Array BioPharma, now owned by Pfizer, and licensed to AstraZeneca in 2003. AstraZeneca and Merck entered into a partnership in 2017 to co-develop and market selumetinib, as well as the PARP inhibitor Lynparza (olaparib), for multiple cancer types.

In the US, selumetinib has breakthrough and orphan drug status, as well as an orphan drug designation in Europe, for the treatment of NF1.  

In 2016, AstraZeneca announced that a Phase III study of selumetinib as a second-line treatment in patients with KRAS mutation-positive locally-advanced or metastatic non-small-cell lung cancer failed to meet its primary endpoint of progression-free survival (PFS), while the drug also had no significant effect on overall survival. An earlier late-stage trial of selumetinib in combination with dacarbazine for the treatment of patients with metastatic uveal melanoma also failed to hit its main goal of PFS.

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