Efficacy of selumetinib

In April 2020The U.S. Food and Drug Administration (FDA) approved selumetinib for use in patients 2 years and older with 1 Neurofibromatosis type 1 (NF1) in pediatric patients with symptomatic, inoperable plexiform neurofibromas (PN). The kinase inhibitor selumetinib is the first approved therapy for pediatric patients with this debilitating and often disfiguring rare disease.

The efficacy of selumetinib was studied in the SPRINT clinical study in the treatment of pediatric patients with NF1 and measurable target PN who were not Ability to be surgically removed without significant risk of morbidity, present in≥20% of patients including disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment, and bladder/bowel dysfunction. The primary efficacy outcome measure is NCI-assessed overall response rate (ORR), defined as the percentage of patients whose tumor volume is reduced by ≥20% as confirmed by magnetic resonance imaging (MRI) within 3-6 months, with an effective rate of 66%. All patients had a partial response, and 82% of responders continued to respond for at least 12 months. An independent central review of ORR using the same response criteria showed an ORR of 44%.

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.