 | |
| Clinical data | |
|---|---|
| Trade names | Gomekli |
| Other names | PD-0325901 |
| AHFS/Drugs.com | Monograph |
| MedlinePlus | a625043 |
| License data | |
| Routes of administration | By mouth |
| Drug class | Antineoplastic |
| ATC code | |
| Legal status | |
| Legal status | |
| Identifiers | |
| CAS Number | |
| PubChemCID | |
| IUPHAR/BPS | |
| DrugBank | |
| ChemSpider | |
| UNII | |
| KEGG | |
| ChEBI | |
| ChEMBL | |
| PDB ligand | |
| CompTox Dashboard(EPA) | |
| ECHA InfoCard | 100.213.070 |
| Chemical and physical data | |
| Formula | C16H14F3IN2O4 |
| Molar mass | 482.198 g·mol−1 |
| 3D model (JSmol) | |
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Mirdametinib, sold under the brand nameGomekli, is amedication used for the treatment of people withneurofibromatosis type 1.[1] Mirdametinib is a kinase inhibitor.[1][4] It is takenby mouth.[1]
The most common adverse reactions in adults include rash, diarrhea, nausea, musculoskeletal pain, vomiting, and fatigue.[5] The most common grade 3 or 4 laboratory abnormalities include increasedcreatine phosphokinase.[5] The most common adverse reactions in children include rash, diarrhea, musculoskeletal pain, abdominal pain, vomiting, headache,paronychia,left ventricular dysfunction, and nausea.[5] The most common grade 3 or 4 laboratory abnormalities include decreasedneutrophil count and increased creatine phosphokinase.[5]
Mirdametinib was approved for medical use in the United States in February 2025.[1][5]
Mirdametinib isindicated for the treatment of people with neurofibromatosis type 1 who have symptomaticplexiform neurofibromas not amenable to complete resection.[1]
The most common adverse reactions in adults include rash, diarrhea, nausea, musculoskeletal pain, vomiting, and fatigue.[5] The most common grade 3 or 4 laboratory abnormalities include increased creatine phosphokinase.[5] The most common adverse reactions in children include rash, diarrhea, musculoskeletal pain, abdominal pain, vomiting, headache, paronychia, left ventricular dysfunction, and nausea.[5] The most common grade 3 or 4 laboratory abnormalities include decreased neutrophil count and increased creatine phosphokinase.[5]
Mirdametinib can cause left ventricular dysfunction and ocular toxicity includingretinal vein occlusion,retinal pigment epithelial detachment, and blurred vision.[5]
The efficacy of mirdametinib was evaluated in ReNeu (NCT03962543), a multicenter, single-arm trial in 114 participants aged two years of age and older (58 adults, 56 pediatric participants) with symptomatic, inoperable NF1-associated plexiform neurofibromas causing significant morbidity.[5] An inoperable plexiform neurofibromas was defined as a plexiform neurofibromas that could not be completely surgically removed without risk for substantial morbidity due to encasement or close proximity to vital structures, invasiveness, or high vascularity.[5]
The USFood and Drug Administration (FDA) granted the application for mirdametinibpriority review,fast track, andorphan drug designations along with a priority review voucher.[5]
Mirdametinib was approved for medical use in the United States in February 2025.[5][6][7]
In May 2025, theCommittee for Medicinal Products for Human Use of theEuropean Medicines Agency adopted a positive opinion, recommending the granting of a conditional marketing authorization for the medicinal product Ezmekly, intended for the treatment of plexiform neurofibromas (PN) in adults and children from two years of age with neurofibromatosis type 1 (NF1).[2] The applicant for this medicinal product is SpringWorks Therapeutics Ireland Limited.[2] Mirdametinib was authorized for medical use in the EU in July 2025.[3]
This article incorporates text from this source, which is in thepublic domain.