SpringWorks Therapeutics

NeurologyLive's Friday 5 highlights: 1) Sharon Cohen on mivelsiran for early-stage Alzheimer disease; 2) Aliza Alter on BHV-7000 for focal epilepsy; 3) Jim Cassidy on mirdametinib for neurofibromatosis type 1; 4) Crystal Proud on SMA research and treatments; 5) Michael Levy on ravulizumab for NMOSD.

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.

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.

SpringWorks Therapeutics completes NDA submission to FDA for mirdametinib, an investigational MEK inhibitor for treating NF1-PN in children and adults. The submission includes data from the Phase 2b ReNeu trial, showing significant response rates, deep and durable responses, and a manageable safety profile. Mirdametinib has received Orphan Drug and Fast Track designations from the FDA and is planned for MAA submission to the EMA in the second half of 2024.