Koselugo司美替尼 selumetinib

Drug Class

MEK1/2 inhibitor

Mechanism

Selumetinib is a selective inhibitor of mitogen-activated protein kinase kinases 1 and 2 (MEK1/2), blocking downstream RAF-MEK-ERK signaling pathway activation in cells with NF1 mutation.

Approved Indication

Treatment of pediatric patients 1 year and older and adult patients with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN).

What This Means For Patients

Koselugo (selumetinib) is a targeted MEK inhibitor approved to treat pediatric and adult patients with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas. It works by blocking the proteins that promote tumor cell growth. It is approved in mainland China and also clinically accessible in the Boao Lecheng pilot zone.

Regulatory Approvals

• FDA — approved (2020) — label
• EMA — approved (2021) — source
• NMPA — approved (2023) — source

Pivotal Clinical Evidence

• SPRINT — Gross et al., N Engl J Med 2020 (NCT01362803) — DOI

Sources

• https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/213756s000lbl.pdf
• https://www.ema.europa.eu/en/medicines/human/EPAR/koselugo
• https://doi.org/10.1056/NEJMoa1912735