Summary

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• Koselugo (selumetinib) is indicated for the treatment of pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN).
• This summary is based on the review of four systematic review(s)/meta-analysis(es). ^[1-4]
• Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN): The pooled Objective Response Rate (ORR) ranged from 68.0% (95% CI 58.0-77.3%) to 73.8% (95% CI 57.3%-85.5%) across 10 clinical trials involving 268 patients and 5 studies involving 126 patients, respectively. The Disease Control Rate (DCR) was reported between 92.5% (95% CI 66.5%-98.7%) and 96.8% (95% CI 90.8-99.7%).
• Non-Small Cell Lung Cancer (NSCLC): The overall efficacy of selumetinib in patients with late-stage/metastatic NSCLC was reported as 71.77% (95% CI: 63.24-81.45%). Selumetinib monotherapy had an efficacy rate of 74.08%, higher than the 65.20% observed with combined therapy.
• Comparisons with Other Drugs (NF1 and PN): Selumetinib showed a partial response rate of approximately 68%-71% for inoperable or progressive plexiform neurofibromas, compared to a 36%-41% partial response rate for bevacizumab in vestibular schwannomas.
• NF1 and PN: Gastrointestinal reactions were common, with diarrhea occurring in 62.5%-63.8% and vomiting in 54.5% of patients. An increase in creatine kinase levels was noted in 63.3% of patients. Most adverse events were mild.
• NSCLC: The overall serious adverse event (SAE) rate was 42.96% (95% CI: 34.74-53.13%). Selumetinib had a significantly better safety profile compared to combined therapy (10.49% vs. 47.38%), with placebo showing the best safety profile.
• There is no population type or subgroups information available in the reviewed studies.