What disease does Gomekli (mirdametinib) treat?

Gomekli (generic name: mirdametinib) is a MEK1/2 inhibitor mainly used to treat plexiform neurofibromas (PN) associated with neurofibromatosis type 1 (NF1). NF1 is a genetic disease in which the RAS/MAPK signaling pathway is abnormally activated in patients, resulting in multiple tumors in the nervous system. Plexiform neurofibromas are a common type of tumor in patients with NF1. They usually occur around nerves and may affect normal tissue, causing pain, deformity, or other serious complications. Since PN is usually difficult to completely remove through surgery, medical treatment becomes an important means of control.

Gomekli blocks the MAPK signaling pathway by inhibiting the activity of MEK1 and MEK2 kinases, thereby reducing the growth and spread of tumor cells. In the clinical trial ReNeu, the drug had a higher overall response rate in patients with NF1-related PN and showed good efficacy. In adult patients, Gomekli's response rate was about 41%, while in pediatric patients the response rate was even higher, at 52%. In addition, Gomekli can reduce pain and dysfunction caused by tumor growth and improve patients' quality of life.

Common side effects of Gomekli include rash, diarrhea, nausea, fatigue, vomiting and musculoskeletal pain. In addition, some patients may experience more serious adverse reactions, such as cardiac dysfunction (such as decreased left ventricular ejection fraction), retinal vein occlusion, or neutropenia. Therefore, patients using this drug need to monitor their heart and vision health regularly during treatment to prevent potentially serious complications.
In February 2025, the US FDA officially approved Gomekli for the treatment of NF1-related plexiform neurofibromas, making it another MEK inhibitor for this disease after Selumetinib. The drug is currently available in the United States, but availability in other countries and regions may vary. For patients with NF1-related PN, especially those whose tumors cannot be surgically removed or whose symptoms are severe, Gomekli provides a new treatment option that has the potential to improve their condition and improve their quality of life.
Reference: https://www.gomekli.com/