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FDA Grants Priority Review to Mirdametinib for NF1-Associated Plexiform Neurofibromas

The FDA has granted priority review to mirdametinib for treating neurofibromatosis type 1–associated plexiform neurofibromas, based on promising phase 2b trial results.

A new drug application (NDA) seeking the approval of mirdametinib in the treatment of adult and pediatric patients with neurofibromatosis type 1–associated plexiform neurofibromas (NF1-PN) has been accepted by the FDA and granted priority review status. The NDA is based on findings from the phase 2b ReNeu trial (NCT03962543) in which the agent induced an objective response rate of 41% per blinded independent central review in adult patients and 52% in children aged 2 years or older who had NF1-PN causing significant morbidity. The application has been assigned an action date of February 28, 2025.
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Reference News

[1]
The OncFive: Top Oncology Articles for the Week of 8/25
onclive.com · Aug 31, 2024

FDA prioritizes mirdametinib for NF1-PN treatment; DESTINY-Breast06 questions HER2 testing necessity for T-DXd in HR+ br...

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