Gomekli FDA Approval History
Last updated by Judith Stewart, BPharm on Feb 11, 2025.
FDA Approved: Yes (First approved February 11, 2025)
Brand name: Gomekli
Generic name: mirdametinib
Dosage form: Capsules and Tablets for Oral Suspension
Company: SpringWorks Therapeutics, Inc.
Treatment for: Neurofibromatosis
Gomekli (mirdametinib) is a MEK inhibitor for the treatment of patients with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN).
- Gomekli is indicated for the treatment of adult and pediatric patients 2 years of age and older with NF1-PN not amenable to complete resection.
- Neurofibromatosis type 1 (NF1) is a rare genetic disorder that arises from mutations in the NF1 gene. The NF1 gene encodes for neurofibromin, a key suppressor of the MAPK pathway which is a key signaling network that regulates cell growth and survival, and plays a central role in multiple oncology and rare disease indications when genetically altered. NF1 patients are at risk of developing plexiform neurofibromas, which are tumors that grow along the peripheral nerve sheath and can cause severe disfigurement, pain and functional impairment. Plexiform neurofibromas can transform into malignant peripheral nerve sheath tumors, which can be aggressive and potentially fatal.
- Gomekli contains mirdametinib, which works by inhibiting mitogen-activated protein kinase kinases 1 and 2 (MEK1/2). MEK1/2 proteins are upstream regulators of the extracellular signal-related kinase (ERK) pathway. In vitro, mirdametinib was shown to inhibit kinase activity of MEK1 and MEK2, and downstream phosphorylation of ERK, which has been shown to reduce neurofibroma tumor volume and proliferation in a mouse model of NF1.
- FDA approval of Gomekli was based on results from the Phase 2b ReNeu trial of 58 adults and 56 pediatric patients with NF1-PN, which showed that Gomekli treatment resulted in a robust objective response rate (ORR), deep and durable reductions in tumor volume, and a manageable safety profile. Gomekli demonstrated a 41% ORR in adults and 52% in children, and the median best percentage change in target tumor volume was -41% (range: -90 to 13%) in adults and -42% (range: -91 to 48%) in children.
- Gomekli capsules and tablets for oral suspension are administered orally twice daily, with or without food, for the first 21 days of each 28-day cycle until disease progression or unacceptable toxicity.
- Warnings and precautions associated with Gomekli include ocular toxicity, left ventricular dysfunction, dermatologic adverse reactions including rash, and embryo-fetal toxicity.
- Common adverse reactions (>25%) in adult patients include rash, diarrhea, nausea, musculoskeletal pain, vomiting, and fatigue. Common Grade 3 or 4 laboratory abnormalities (>2%) include increased creatine phosphokinase.
Common adverse reactions (>25%) in pediatric patients include rash, diarrhea, musculoskeletal pain, abdominal pain, vomiting, headache, paronychia, left ventricular dysfunction, and nausea. Common Grade 3 or 4 laboratory abnormalities (>2%) include decreased neutrophil count and increased creatine phosphokinase.
Review the Gomekli Package Insert for more detailed information about this medicine. The Gomekli Package Insert contains more comprehensive information on Indications and Usage, Dosage and Administration, Clinical Pharmacology, Clinical Studies, Drug Interaction, and more.
Development timeline for Gomekli
Further information
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