AstraZeneca, Merck's Koselugo gets approval in Japan for rare genetic disorder in kids
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AstraZeneca (NASDAQ:AZN) and Merck's (NYSE:MRK) Koselugo was approved in Japan to treat patients three years of age and older with plexiform neurofibromas (PNs) in neurofibromatosis type 1 (NF1) with clinical symptoms, such as pain and disfigurement, and PNs which cannot be completely removed by surgery without risk of substantial morbidity.
NF1 is a genetic disorder which causes tumors to grow along the nerves and generally affects the skin. AstraZeneca noted that the condition affects one in 3,000 individuals worldwide.
Koselugo (selumetinib) is the first medicine approved in Japan to treat this rare condition, the British pharma giant said in a Sept. 27 press release.
The approval by Japan's Ministry of Health, Labour and Welfare (MHLW) was backed by data from a phase 2 trial called SPRINT Stratum 1 and a phase 1 study in Japanese pediatric patients with NF1.
"As the first medicine approved in Japan for paediatric patients with symptomatic, inoperable plexiform neurofibromas in neurofibromatosis type 1, Koselugo offers new hope for patients and families affected by this incurable genetic disease, whose only previous treatment option was repeated surgery," said Alexion CEO Marc Dunoyer.
Koselugo is already approved in the U.S. and EU to treat certain patients with NF1 and PNs, the company added.