AstraZeneca's Selumetinib (Koselugo) Approved in China for Pediatric NF1-Associated Plexiform Neurofibromas

On May 8, the website of the National Medical Products Administration (NMPA) showed that AstraZeneca's MEK1/2 inhibitor, selumetinib, was approved for marketing in China. It is indicated for the treatment of patients aged 3 years and above with symptomatic and/or progressive, inoperable neurofibromatosis type 1 (NF1) associated plexiform neurofibromas (PN).

Selumetinib, developed by Array BioPharma (a Pfizer subsidiary), is a mitogen-activated protein kinase kinase 1 and 2 (MEK1/2) inhibitor. It was first approved for marketing in April 2020 under the brand name Koselugo. In December 2003, AstraZeneca entered into an agreement with Array BioPharma to acquire global rights to selumetinib. In July 2017, AstraZeneca partnered with Merck to jointly oversee the global development and commercialization of selumetinib.

The FDA previously approved selumetinib based on the results of a Phase II clinical trial. The trial included a total of 50 patients and aimed to evaluate the efficacy and safety of selumetinib in treating pediatric NF1 patients who are ineligible for surgery. The primary endpoint of the trial was the objective response rate (ORR).

The results showed that the ORR of patients receiving twice-daily selumetinib treatment reached 66%, with all patients achieving partial response. Additionally, 82% of the patients had a duration of response lasting 12 months or longer.

In December 2021, AstraZeneca initiated a Phase III clinical trial (KOMET) of selumetinib in China to further evaluate the safety and efficacy of selumetinib. The trial enrolled 146 patients and is expected to be completed in November 2023.

PN is a type of tumor that involves the nerve sheath (the coating around nerve fibers) and can grow in any part of the body. NF1 is a rare progressive disease caused by mutations or defects in specific genes. NF1 is typically diagnosed in early childhood, with an estimated incidence of 1/3000.

To date, four MEK inhibitors have been approved globally: Selumetinib (AstraZeneca/MSD), Binimetinib (Pfizer/Pierre Fabre/Ono Pharmaceutical), Cobimetinib (Roche/Exelixis), and Trametinib (Japan Tobacco/Novartis). Among them, Trametinib was launched in China in December 2019. Chinese pharmaceutical companies that have invested in MEK inhibitors include Kelun Pharmaceuticals, Hengrui Medicine, Fosun Pharma, and Zhengda Tianqing. Among them, Kelun Pharmaceuticals' Tolerametinib has already filed for marketing authorization in China on January 29 this year, while Hengrui Medicine's SHR7390 and Fosun Pharma's FCN-159 are currently in Phase II clinical trials.

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