European Commission grants conditional approval selumetinib (Koselugo) for treatment of children with symptomatic, inoperable plexiform neurofibromas (PN) and neurofibromatosis type 1 (NF1)

Approval of this inhibitor of MEK1/2 is based on results from SPRINT Stratum 1 phase II trial, which showed selumetinib reduced size of inoperable tumours in children (objective response rate 66%) and also reduced pain and improved quality of life

SPS commentary:

This the first medicine approved in the EU for patients with neurofibromatosis type 1, a rare genetic condition that affects one in 3,000 individuals globally. In 30-50% of people with NF1, tumours develop in the nerve sheaths which can cause disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment and bladder or bowel dysfunction.

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