Selumetinib for the treatment of neurofibromatosis

Selumetinib is the first drug approved for children with neurofibromatosis type 1 and inoperable symptomatic plexiform neurofibromas (PN). To maintain tumor response, treatment with selumetinib is often continued for many years, and long-term safety and efficacy data are unclear. A Phase 1/2 clinical trial of selumetinib in children with inoperable PN provides up to 5 years of additional safety and efficacy data. Overall,

Both tumor response to selumetinib and improvement in tumor-related pain were sustained for those patients who continued treatment. No new safety signals were identified; however, known selumetinib-related adverse events can occur years after treatment initiation, and through 48 cycles of selumetinib treatment, patient- and observer-reported pain outcome measures showed sustained improvements in tumor-related pain intensity and pain interference for those who continued treatment. Therefore, continuous monitoring is required while the patient continues taking the medication. These results indicate that treatment of children with symptomatic, inoperable neurofibroma-associated plexiform neurofibromas in children is safe, tolerable, and generally provides durable clinical benefit.

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.