GOMEKLI (mirdametinib) is a MEK inhibitor that has received Food and Drug Administration (FDA) approval for the treatment of symptomatic neurofibromatosis type 1 (NF1) with plexiform neurofibromas (PN). It is recommended for adults and pediatric patients aged 2 years and older who have tumors that cannot be completely resected. A Priority Review Voucher (PRV) for pediatric diseases was granted to SpringWorks in recognition of this novel treatment approach. NF1-PN is a rare genetic disorder affecting approximately 100,000 in the U.S. Of these, 40,000 adults currently have no approved treatment options.
SpringWorks CEO Saqib Islam highlighted the NF1-PN patient community needs additional treatment choices. He expressed gratitude for the opportunity to serve both adults and children with NF1-PN because this approval brings a therapy option to reduce tumors and bring significant effects to improve symptoms.
Dr. Christopher Moertel, lead investigator of the ReNeu trial and Medical Director of the Pediatric Neuro-Oncology and Neurofibromatosis Programs at the University of Minnesota, hailed this approval as a game-changer. Patients with NF1-PN often face significant health challenges, with historically having limited treatment options. He expressed confidence in the ReNeu trial results, stating that GOMEKLI demonstrated strong and lasting benefits and enabled the patients to continue treatment.
It was assessed in the Phase 2b ReNeu trial, which included 114 patients with NF1-PN (58 adults and 56 children). The results showed strong efficacy with an objective response rate (ORR) of 41% in adults and 52% in children, determined by a blinded independent central review. The therapy resulted in significant long-lasting reductions of tumors in both adult and pediatric patients through the peak median reductions of -41% and -42%, respectively. The therapy delivered long-term pain relief and a high quality of life.
Around 88% of adult and 90% of pediatric participants maintained their responses for more than one year throughout the study period. Additionally, 50% of adults, along with 48% of children, sustained the duration response for more than twenty-four months. Both groups achieved substantial early and prolonged improvements.
This drug has achieved a manageable safety profile with common adverse effects in adults and pediatrics. The FDA approval also included safety warnings regarding left ventricular dysfunction, ocular toxicity, dermatologic adverse reactions, and embryo-fetal toxicity, it granted GOMEKLI Priority Review and previously designated it as an Orphan drug and Fast Track therapy for NF1-PN. This priority review voucher awarded to SpringWorks underscores the FDA’s commitment to expediting the development of treatments for rare pediatric diseases.
SpringWorks supports the services for patients with NF1-PN, including insurance coverage, financial aid, and educational support through its Care Connections program. GOMEKLI is available as capsules and tablets in the US within two weeks. SpringWorks’ Marketing Authorization Application for mirdametinib is currently under review, with a decision expected in 2025.
Reference: SpringWork Therapeutics. SpringWorks Therapeutics Announces FDA Approval of GOMEKLI™ (mirdametinib) for the Treatment of Adult and Pediatric Patients with NF1-PN. Published February 11, 2025. Accessed February 12, 2025. SpringWorks Therapeutics Announces FDA Approval of GOMEKLI™ (mirdametinib) for the Treatment of Adult and Pediatric Patients with NF1-PN | SpringWorks Therapeutics
