FDA Grants Priority Review to New Drug Application of Neurofibromatosis Agent Mirdametinib
The FDA has granted priority review to SpringWorks Therapeutics' NDA for mirdametinib, an investigational MEK inhibitor for treating NF1-PN. The European Medicines Agency also validated the MAA for the same indication. Data from the ReNeu trial showed significant reductions in PN volume and improvements in pain and quality of life. SpringWorks anticipates potential launch in early 2025.
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SpringWorks' CEO, Saqib Islam, expresses optimism about delivering a transformative medicine for NF1-PN in the U.S. and Europe, highlighting the need for new treatment advances. The FDA and EMA have granted various designations for mirdametinib, including Priority Review, Orphan Drug, Fast Track, and Rare Pediatric Disease, indicating its potential significance. The ReNeu trial results showed mirdametinib's efficacy in reducing tumor volume and improving quality of life with a manageable safety profile. Annette Bakker of the Children's Tumor Foundation congratulates SpringWorks on this milestone, emphasizing the urgent need for more treatment options for NF1 patients.
The FDA has granted priority review to SpringWorks Therapeutics' NDA for mirdametinib, an investigational MEK inhibitor for treating NF1-PN. The European Medicines Agency also validated the MAA for the same indication. Data from the ReNeu trial showed significant reductions in PN volume and improvements in pain and quality of life. SpringWorks anticipates potential launch in early 2025.