AstraZeneca's Selumetinib Capsules Recommended for CDE Priority Review as First-in-Class Treatment for Pediatric NF1-Associated Plexiform Neurofibromas

Today, the CDE official website announced that AstraZeneca's Selumetinib capsules are proposed to be included in the priority review. Selumetinib is the world's first approved drug for the treatment of plexiform neurofibromas (PN) in pediatric practice for Neurofibromatosis Type 1.

Selumetinib is an oral selective inhibitor of the MEK enzyme, a component of the mitogen-activated protein kinase (MAPK) signaling pathway, which can block this enzyme to inhibit cell growth and proliferation. The drug demonstrated high efficacy in the SPRINT clinical trial for children aged 2 to 18 with NF1 and inoperable PN. Selumetinib not only reduced tumor volume in some children but also alleviated pain and improved their quality of life.

In June 2021, the European Commission (EC) granted conditional approval to Selumetinib for the treatment of symptomatic, inoperable plexiform neurofibromas in pediatric patients aged 3 years and older with Neurofibromatosis Type 1 (NF1).