Mapkure, LLC

• The FDA has approved Gomekli (mirdametinib) for both adult and pediatric patients with Neurofibromatosis Type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection. • Approval was based on Phase 2b ReNeu trial data, demonstrating a 41% ORR in adults and 52% in children, alongside deep and durable tumor volume reductions and manageable safety profiles. • Gomekli is the first and only medicine approved for both adults and children with NF1-PN, addressing a significant unmet need, particularly for adult patients who previously lacked approved treatments. • SpringWorks received a rare pediatric disease priority review voucher from the FDA, potentially expediting the review of future drug candidates.

FDA Approves SpringWorks' Gomekli (Mirdametinib) for Neurofibromatosis Type 1