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Gomekli

mirdametinib
MEK1/2 InhibitorFDA Approved 2025Day One Biopharmaceuticals
Route
Oral
Half-Life
~6 hrs
FDA Approved
2025
Manufacturer
Day One Biopharmaceuticals
1. Indications and Usage

Adult and pediatric patients 2 years and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas.

2. Dosage and Administration

Adults: 2 mg orally twice daily. Pediatric patients: dosed by body surface area. Continue until disease progression or unacceptable toxicity.

3. Dosage Forms and Strengths

Capsules: 2 mg; Oral Solution: 0.4 mg/mL

4. Contraindications

None listed in the prescribing information.

5. Warnings and Precautions
  • Cardiomyopathy: Decreased LVEF reported. Assess LVEF at baseline and periodically.
  • Ocular Toxicity: Retinal pigment epithelial detachment reported. Ophthalmologic exams recommended.
  • Rash: Severe rash including acneiform dermatitis reported.
  • Embryo-Fetal Toxicity: Can cause fetal harm.
6. Adverse Reactions
Most Common Adverse Reactions

Dermatitis Acneiform (72%), Nausea (42%), Vomiting (36%), Diarrhea (34%), Paronychia (30%), Fatigue (28%), Dry Skin (24%), Increased CPK (20%)

Dermatitis Acneiform
72%
Nausea
42%
Vomiting
36%
Diarrhea
34%
Paronychia
30%
Fatigue
28%
Dry Skin
24%
Increased CPK
20%

Consult the complete prescribing information for a comprehensive list of adverse reactions and their frequencies.

7. Drug Interactions

Strong CYP1A2 Inhibitors: Avoid; may increase mirdametinib exposure.
Strong CYP1A2 Inducers: Avoid; may reduce efficacy.

8. Use in Specific Populations
Pregnancy

Consult the full prescribing information for pregnancy-related considerations.

Lactation

Refer to prescribing information for lactation guidance.

Pediatric Use

Pediatric safety and efficacy information is detailed in the full label.

Hepatic/Renal Impairment

Dose modifications for organ impairment are specified in the complete prescribing information.

12. Clinical Pharmacology
Mechanism of Action

Mirdametinib is a highly selective, non-ATP-competitive allosteric inhibitor of MEK1/MEK2. Inhibition of MEK in the RAS-MAPK pathway reduces proliferation of neoplastic cells in plexiform neurofibromas driven by NF1 loss-of-function mutations.

Pharmacokinetics

Tmax: 1-3 hours. Protein binding: ~97%. Metabolized by CYP1A2 and UGTs. Half-life: ~5 hours. Elimination: feces 76%, urine 18%.

14. Clinical Studies

Clinical efficacy and safety data supporting the approval are available in the full prescribing information and from the clinical trials listed below.

Pivotal Clinical Trials
Additional Resources
FDA-Approved Tumor Types

Gomekli has FDA-approved indications across the following cancer types covered on CancerDrugEvidence:

External Resources
Important Notice: This page is intended as a navigational reference to the FDA-approved prescribing information for Gomekli. It does not replace the full prescribing information. Healthcare professionals should consult the complete package insert available at DailyMed before making prescribing decisions. Patient-specific factors should always guide clinical decision-making.

Frequently Asked Questions

What is Gomekli (mirdametinib) approved for?

Gomekli (mirdametinib) is an FDA-approved oncology agent. Refer to the full prescribing information for complete indication details.

How is Gomekli (mirdametinib) administered?

Refer to the full prescribing information for dosing schedules, administration instructions, and dose modifications.

How does Gomekli (mirdametinib) work?

Mirdametinib is a highly selective, non-ATP-competitive allosteric inhibitor of MEK1/MEK2. Inhibition of MEK in the RAS-MAPK pathway reduces proliferation of neoplastic cells in plexiform neurofibromas driven by NF1 loss-of-function mutations.

What are the most common side effects?

Dermatitis Acneiform (72%), Nausea (42%), Vomiting (36%), Diarrhea (34%), Paronychia (30%), Fatigue (28%), Dry Skin (24%), Increased CPK (20%) Dermatitis Acneiform 72% Nausea 42% Vomiting 36% Diarrhea 34% Paronychia 30% Fatigue 28% Dry Skin 24% Increased CPK 20%