Mirdametinib

【Indications】

GOMEKLI is indicated for the treatment of adult and pediatric patients aged 2 years and older with neurofibromatosis type 1 (NF1) and symptomatic plexiform neurofibromas (PN) that are not amenable to complete surgical resection.

【Recommended Dosage】

• Standard Dose: 2 mg/m² orally twice daily (approximately every 12 hours), with or without food. Each cycle is 28 days (21 days on treatment followed by 7 days off). Continue treatment until disease progression or unacceptable toxicity.

• Maximum Dose: 4 mg twice daily.

【Adverse Reactions】

Adults:

• The most common adverse reactions (incidence >25%) were rash, diarrhea, nausea, musculoskeletal pain, vomiting, and fatigue.

• The most common Grade 3 or 4 laboratory abnormalities (incidence >2%) were increased creatine phosphokinase.

Pediatric Patients:

• The most common adverse reactions (incidence >25%) were rash, diarrhea, musculoskeletal pain, abdominal pain, vomiting, headache, paronychia, left ventricular dysfunction, and nausea.

• The most common Grade 3 or 4 laboratory abnormalities (incidence >2%) were decreased neutrophil count and increased creatine phosphokinase.

【Mechanism of Action】

Mirdametinib is an inhibitor of MEK1/2 (mitogen-activated protein kinase kinase). MEK1/2 proteins serve as upstream regulators of the extracellular signal-related kinase (ERK) pathway. In vitro, mirdametinib inhibits the kinase activity of MEK1 and MEK2, as well as downstream phosphorylation of ERK.

【Storage】

Store capsules and tablets for oral suspension at 20°C to 25°C, with permitted excursions between 15°C and 30°C. Protect from light.