LONDON (ShareCast) - (Sharecast News) - The US Food and Drug Administration has approved the expanded usage of AstraZeneca's Koselugo drug to adults with neurofibromatosis type 1 (NF1) - a rare, progressive, genetic condition that can impact every organ system.
Koselugo, also known as selumetinib, is an oral treatment that inhibits the mitogen-activated protein kinase enzymes, which in up to 50% of NF1 patients can lead to non-malignant tumours called plexiform neurofibromas (PN) that may affect the brain, spinal cord and nerves.

Following the FDA approval, Koselugo can now be used to treat adult patients with NF1 in the US who have symptomatic, inoperable PN, expanding its usage from just "certain paediatric patients" previously.

The FDA development was based on the KOMET Phase III trial, the largest and only placebo-controlled global Phase III trial in this patient population, which showed a 20% overall response rate in tumour size reduction.

Marc Dunoyer, head of AstraZeneca's rare disease division Alexion, said Koselugo has already "transformed the treatment standard for this rare disease", and the approval "enables much-needed continuity of care and supports patients across the disease journey in the US".

Meanwhile, Annette Bakker, head of the Children's Tumor Foundation, said the approval was a "major step forward for NF patients everywhere", adding: "Koselugo has already changed what is possible for children with NF1, and now adults will benefit from that same progress."

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