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FDA Grants Priority Review to New Drug Application of Neurofibromatosis Agent Mirdametinib

According to a new announcement, the FDA has accepted and granted priority review to SpringWorks Therapeutics’ new drug application (NDA) for mirdametinib, an investigational MEK inhibitor for the treatment of adult and pediatric patients with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN). The agency has scheduled a PDUFA action date of February 28, 2025, and stated that it does not have current plans to hold an advisory committee meeting to discuss the application.

In the update, the company also announced that the European Medicines Agency validated the Marketing Authorization Application (MAA) for mirdametinib for the treatment of adult and pediatric patients with NF1-PN. Both submissions for the NDA and the MAA included data from the pivotal phase 2b ReNeu trial (NCT03962543), a multicenter, open-label study that featured 114 patients with NF1 PN (58 adult and 56 pediatric) who received mirdametinib for up to 24 months.

Recently presented data from ReNeu at the 2024 American Society of Clinical Oncology Annual Meeting showed that treatment with mirdametinib resulted in statistically significant objective response rate (ORR) and sustained reductions in PN volume. Coupled with improvements in pain severity, pain interference, and health-related quality of life (HRQoL), results showed that the tablet therapy may be a potentially therapeutic option for patients with NF1 PN across all ages.

Mirdametinib has shown promising results in reducing tumor volume and improving the quality of life for both adult and pediatric patients with NF1-PN. The FDA’s priority review and the European Medicines Agency's validation highlighted the drug's potential impact as a new treatment option for NF1-PN. While mirdametinib is progressing through regulatory channels, its safety profile and observed adverse effects will be critical factors in determining its widespread adoption.

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