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) [3][5][17] - The approval is based on positive results from the Phase 2b ReNeu trial, demonstrating significant efficacy and a manageable safety profile [6][8][9] - The company has been granted a rare pediatric disease priority review voucher by the FDA, highlighting the importance of this treatment for the NF1-PN patient community [3][6] Company Overview - SpringWorks Therapeutics is a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer [27] - The company has developed GOMEKLI as the first FDA-approved medicine for NF1-PN, and it also markets OGSIVEO (nirogacestat) for adults with desmoid tumors [27] - SpringWorks is advancing a diverse portfolio of targeted therapy candidates for solid tumors and hematological cancers [27] Product Details - GOMEKLI is an oral MEK inhibitor approved for patients aged 2 years and older with symptomatic plexiform neurofibromas not amenable to complete resection [17][12] - The product is available in 1 mg and 2 mg capsules, as well as a 1 mg tablet for oral suspension [12] - GOMEKLI is expected to be available through specialty pharmacies and distributors in the U.S. within two weeks of approval [12] Clinical Trial Insights - The Phase 2b ReNeu trial enrolled 114 patients (58 adults and 56 pediatric patients) and met its primary endpoint with a confirmed objective response rate (ORR) of 41% in adults and 52% in children [7][8] - Tumor volume reductions were significant, with a median best percentage change of -41% in adults and -42% in children [8] - The trial also reported improvements in pain and quality of life for patients, indicating the treatment's potential to enhance overall well-being [8] Market Context - NF1 is a genetic disorder affecting approximately 100,000 individuals in the U.S., with a 30-50% lifetime risk of developing plexiform neurofibromas [5][14] - Prior to GOMEKLI, there were no approved treatments for NF1-PN, making this approval a significant advancement for the patient community [5][6] - The approval of GOMEKLI represents a critical new option for NF patients and their families, addressing a substantial unmet medical need [10][5]