UPDATE: SpringWorks Therapeutics Announces FDA Approval of GOMEKLI™ (mirdametinib) for the Treatment of Adult and Pediatric Patients with NF1-PN

Core Insights - SpringWorks Therapeutics has received FDA approval for GOMEKLI™ (mirdametinib), the first and only treatment for both adults and children with neurofibromatosis type 1 (NF1) associated plexiform neurofibromas (PN) [1][2][11] - The approval is based on positive results from the Phase 2b ReNeu trial, which demonstrated significant tumor volume reductions and a manageable safety profile [3][4][5] Company Overview - SpringWorks Therapeutics is a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, with GOMEKLI being its second FDA approval in less than 18 months [2][21] - The company is also advancing a diverse portfolio of novel targeted therapy product candidates for solid tumors and hematological cancers [21] Product Details - GOMEKLI is an oral MEK inhibitor approved for patients aged 2 years and older with symptomatic NF1-PN not amenable to complete resection [11][21] - The drug is available in 1 mg and 2 mg capsules, as well as a 1 mg tablet for oral suspension, and is expected to be available through specialty pharmacies within two weeks [7][21] Clinical Trial Results - The ReNeu trial enrolled 114 patients (58 adults and 56 pediatric patients) and achieved a confirmed objective response rate (ORR) of 41% in adults and 52% in children [3][4] - The median best percentage change in target PN volume was -41% in adults and -42% in children, with 88% of adults and 90% of children showing a response lasting at least 12 months [3][5] Market Need - NF1 affects approximately 100,000 individuals in the U.S., with about 40,000 living with NF1-PN, highlighting a significant unmet need for effective treatment options [2][9] - Prior to GOMEKLI, there were no approved medications for NF1-PN, making this approval a critical advancement for patients [2][5] Regulatory Status - GOMEKLI was granted a rare pediatric disease priority review voucher by the FDA and has previously received Orphan Drug and Fast Track designations [4][5] - The Marketing Authorization Application for GOMEKLI is under review by the European Medicines Agency (EMA), with a decision expected in 2025 [8]