AstraZeneca, Merck's Koselugo gets approval in China for rare genetic disorder in kids
Leon Neal

AstraZeneca (NASDAQ:AZN) and Merck's (NYSE:MRK) Koselugo was approved in China to treat symptomatic, inoperable plexiform neurofibromas (PN) in children with neurofibromatosis type 1 (NF1) aged three years and above.
NF1 is a genetic disorder which causes tumors to grow along the nerve sheaths (plexiform neurofibromas) and can cause disfigurement, motor dysfunction, pain, airway dysfunction, visual and bladder or bowel issues. The condition affects one in 3,000 individuals globally, AstraZeneca noted.

The approval by China's National Medical Products Administration (NMPA) was backed by data from a phase 2 trial called SPRINT Stratum 1.
AstraZeneca said Koselugo (selumetinib) is the first and only approved therapy in China for this rare genetic condition which can cause growth of benign tumors along nerves in the body.
Koselugo is already approved in the U.S., EU, Japan and several other countries.