Drug updated on 3/28/2024
| Dosage Form | Capsule (oral: 10 mg, 25 mg) |
| Drug Class | Kinase inhibitors |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- For the treatment of pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN).
Summary
- Selumetinib (Koselugo) 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.
- Two systematic reviews/meta-analyses were reviewed to gather information about this drug's efficacy and safety profile.
- A review involving five studies with a total of 126 patients found that selumetinib had an objective response rate (ORR) of 73.8% and disease control rate (DCR) was at 92.5%, indicating its effectiveness in treating NF1 in children.
- The same study also reported common adverse events associated with selumetinib use, including diarrhea occurring at a pooled rate of approximately 63.8% and increased creatine kinase levels observed at around the same frequency.
- Another systematic review focused on non-small cell lung cancer showed that while not superior to combined therapy regarding efficacy, selumetinib demonstrated better safety profile when used alone compared to combination therapies; it had an overall serious adverse event rate estimated at roughly 42%.
- Despite these findings suggesting potential benefits from using Selumetinib as monotherapy or part of combination treatments for different conditions, both reviews emphasized the need for larger-scale randomized controlled trials to confirm long-term outcomes related to this drug's usage due its current evidence being limited or having varying quality across studies included within each analysis respectively.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Koselugo (selumetinib) Prescribing Information. | 2021 | AstraZeneca Pharmaceuticals LP, Wilmington, DE |
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source |
|---|---|---|
| Efficacy and safety of selumetinib in pediatric patients with neurofibromatosis type 1. | 2022 | American Academy of Neurology |
| The efficacy and safety of selumetinib as secondary therapy for late-stage and metastatic non-small cell lung cancer: results from a systematic review and meta-analysis. | 2022 | Annals of Translational Medicine |
| Assessment report: Koselugo. | 2021 | EMA |