CHMP recommends conditional approval for Koselugo to treat paediatric patients with neurofibromatosis type 1 (NF1) plexiform neurofibromas .- AstraZeneca

The Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a conditional marketing authorisation for the medicinal product Koselugo, from AstraZeneca and intended for the treatment of paediatric patients with neurofibromatosis type 1 (NF1) plexiform neurofibromas (PN). Koselugo will be available as 10 mg and 25 mg hard capsules. The active substance of Koselugo is selumetinib, a selective inhibitor of mitogen activated protein kinase kinases 1 and 2 (MEK 1/2) (ATC code: L01EE04). MEK inhibition can block the proliferation and survival of tumour cells in which the RAF-MEK-ERK pathway is activated. The benefits of Koselugo are its ability to reduce the volume of plexiform neurofibromas and thereby improve clinical outcomes such as pain intensity, mobility and/or disfigurement in patients. The most common side effects are vomiting, rash, increased blood creatine phosphokinase, diarrhoea, nausea, dry skin, asthenic events, pyrexia, acneiform rash, hypoalbuminaemia, stomatitis, increased aspartate aminotransferase and paronychia.