Mirdametinib

Mirdametinib, sold under the brand name Gomekli, is a medication used for the treatment of people with neurofibromatosis type 1.^[1] Mirdametinib is a kinase inhibitor.^[1][2] It is taken by mouth.^[1]

Mirdametinib

Clinical data
Trade names	Gomekli
Other names	PD-0325901
AHFS/Drugs.com	Monograph
MedlinePlus	a625043
License data	US DailyMed: Mirdametinib
Routes of administration	By mouth
Drug class	Antineoplastic
ATC code	L01EE05 (WHO)
Legal status
Legal status	US: ℞-only[1]
Identifiers
CAS Number	391210-10-9
PubChem CID	9826528
IUPHAR/BPS	7935
DrugBank	DB07101
ChemSpider	10814340
UNII	86K0J5AK6M
KEGG	D11675
ChEBI	CHEBI:9826528
ChEMBL	ChEMBL507361
PDB ligand	4BM (PDBe, RCSB PDB)
CompTox Dashboard (EPA)	DTXSID0044024
ECHA InfoCard	100.213.070
Chemical and physical data
Formula	C16H14F3IN2O4
Molar mass	482.198 g·mol−1
3D model (JSmol)	Interactive image
SMILES O=C(NOCC(O)CO)C1=CC=C(F)C(F)=C1NC2=CC=C(I)C=C2F
InChI InChI=1S/C16H14F3IN2O4/c17-11-3-2-10(16(25)22-26-7-9(24)6-23)15(14(11)19)21-13-4-1-8(20)5-12(13)18/h1-5,9,21,23-24H,6-7H2,(H,22,25)/t9-/m1/s1 Key:SUDAHWBOROXANE-SECBINFHSA-N

The most common adverse reactions in adults include rash, diarrhea, nausea, musculoskeletal pain, vomiting, and fatigue.^[3] The most common grade 3 or 4 laboratory abnormalities include increased creatine phosphokinase.^[3] The most common adverse reactions in children include rash, diarrhea, musculoskeletal pain, abdominal pain, vomiting, headache, paronychia, left ventricular dysfunction, and nausea.^[3] The most common grade 3 or 4 laboratory abnormalities include decreased neutrophil count and increased creatine phosphokinase.^[3]

Mirdametinib was approved for medical use in the United States in February 2025.^[1][3]

Medical uses

Mirdametinib is indicated for the treatment of people with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas not amenable to complete resection.^[1]

Adverse effects

The most common adverse reactions in adults include rash, diarrhea, nausea, musculoskeletal pain, vomiting, and fatigue.^[3] The most common grade 3 or 4 laboratory abnormalities include increased creatine phosphokinase.^[3] The most common adverse reactions in children include rash, diarrhea, musculoskeletal pain, abdominal pain, vomiting, headache, paronychia, left ventricular dysfunction, and nausea.^[3] The most common grade 3 or 4 laboratory abnormalities include decreased neutrophil count and increased creatine phosphokinase.^[3]

Mirdametinib can cause left ventricular dysfunction and ocular toxicity including retinal vein occlusion, retinal pigment epithelial detachment, and blurred vision.^[3]

History

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.^[3] 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.^[3]

The US Food and Drug Administration (FDA) granted the application for mirdametinib priority review, fast track, and orphan drug designations along with a priority review voucher.^[3]

Society and culture

Legal status

Mirdametinib was approved for medical use in the United States in February 2025.^[3][4][5]

In May 2025, the Committee for Medicinal Products for Human Use of the European 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).^[6] The applicant for this medicinal product is SpringWorks Therapeutics Ireland Limited.^[6]