What is selumetinib?

The U.S. Food and Drug Administration (FDA) approved selumetinib for the treatment of pediatric patients 2 years and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable tumors. Plexiform neurofibromas (PN) are one of the most striking clinical features of neurofibromatosis1 and occur mainly in childhood and during hormonal changes. There are no previously approved medical therapies for tumor shrinkage or regression, and surgery is not always possible due to inaccessible location, involvement of vital tissue, optimal timing, and incomplete resection.

Neurofibromin is a long cytoplasmic protein of 2818 amino acids that is the product of the neurofibromatosis type 1 gene. It inhibits the activity of Ras GTPase protein. Lack of functional neurofibromin in patients with NF1 leads to Ras dysregulation and tumorigenesis. The RAS MAPK pathway is highly activated in NF1. Selumetinibis an inhibitor of MEK1 and MEK2 proteins, which play an important role in the MAPK signaling pathway related to tumor growth. Approval was based on a pivotal single-arm Phase II trial. Seventy percent of participants experienced a confirmed partial response of tumor shrinkage, and 68% also experienced improvements in related complications. Other studies have also shown beneficial responses.

SelumetinibThe original drug has been launched in China, but it has not yet entered the scope of medical insurance. SpecificationThe price of 10mg*60 tablets per box may be more than 20,000 yuan, which is relatively expensive. Selumetiniboriginal drug marketed overseas, specifications10mg*60 capsules per box may cost more than RMB 50,000 (the price may fluctuate due to exchange rates). The ingredients of the original selumetinib sold domestically and abroad are basically the same. There is currently no generic version of selumetinib produced and marketed. For more drug information and specific prices, please consult the medical consultant of Yaode.