What does selumetinib treat
Selumetinib is a MEK1/2 inhibitor targeted drug primarily used to treat type 1 neurofibromatosis (NF1) - related plexiform neurofibromatosis. It inhibits tumor growth by blocking the MAPK signaling pathway and is the first oral targeted drug approved for this indication.
1. Basic information of the drug
(1) Common name: Smetinib
(2) Product Name: Koselugo
(3) Drug category: MEK1/2 inhibitor
2 main treatment indications
(1) Type 1 neurofibromatosis: suitable for symptomatic, non-surgical treatment of plexiform neurofibromatosis in NF1 patients aged 2 years and above.
(2) Clinical trial indications: We are currently studying solid tumors with various RAS/RAF pathway abnormalities, including low-grade serous ovarian cancer, thyroid cancer, etc.
3 Pharmacological mechanisms of action
(1) Target inhibition: Selective inhibition of MEK1 and MEK2 kinase activity, blocking the MAPK signaling pathway.
(2) Antitumor effect: By inhibiting the abnormal activation of this pathway, it reduces tumor cell proliferation and induces apoptosis.
4 Clinical efficacy characteristics
(1) Tumor response rate: Key trials have shown an overall response rate of 66%, which can sustainably reduce tumor volume.
(2) Symptom improvement: It can significantly alleviate tumor related symptoms such as pain and motor dysfunction.
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