Koselugo approved in US for adults with neurofibromatosis type 1

The US Food and Drug Administration (FDA) has approved Koselugo (selumetinib) for the treatment of adult patients with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN). The approval was granted to Alexion, AstraZeneca Rare Disease.

The regulatory decision is based on positive results from the KOMET Phase III trial, which is the largest and only placebo-controlled global study for this patient population. The trial demonstrated a significant tumor size reduction in patients treated with Koselugo.

Neurofibromatosis type 1 (NF1) is a rare, progressive genetic disorder that can affect multiple organ systems. Plexiform neurofibromas (PN), which can cause debilitating symptoms such as pain and muscle weakness, develop in up to 50% of individuals with NF1.

This expanded approval allows for continuity of care for NF1 patients across different age groups in the US, as Koselugo is also approved for pediatric patients with NF1 PN.