The Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) and granted priority review to selumetinib for patients ≥3 years with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas. 

Selumetinib is an oral mitogen activated protein kinase (MEK) 1/2 inhibitor being co-developed by AstraZeneca and Merck. The NDA includes data from the phase 2 SPRINT trial that evaluated twice-daily oral monotherapy with selumetinib.

Results showed an objective response rate (defined as percentage of patients with a confirmed complete or partial response of ≥20% tumor volume reduction) was achieved in 66% (n=33/50) of patients. 

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Selumetinib was previously granted Orphan Drug and Breakthrough Therapy designation for the same population. A Prescription Drug User Fee Act date has been set for the second quarter of 2020.


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For more information visit Merck.com.