Gomekli Application Scope

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

Characteristics

Ingredients:
- Active: Mirdametinib
- Inactive (capsules): Gelatin, titanium dioxide, FD&C Blue #1, yellow iron oxide, among others
- Inactive (tablets): Microcrystalline cellulose, croscarmellose sodium, magnesium stearate, grape flavor, sucralose

Properties: MEK1/2 kinase inhibitor; reduces tumor volume and proliferation in NF1-associated plexiform neurofibromas
Packaging Specification: Capsules and tablets supplied in bottles of 42 or 84 units

Storage:
- Store at 20°C to 25°C (68°F to 77°F)
- Excursions permitted between 15°C and 30°C (59°F to 86°F)
- Protect from light
Expiry Date: Refer to the product packaging for the expiration date
Executive Standard: Refer to FDA-approved labeling and prescribing information
Approval Number: FDA-approved in February 2025
Date of Revision: February 2025
Manufacturer: SpringWorks Therapeutics, Inc.

Guidelines for the Use of Gomekli

Dosage and Administration:
- Recommended dose: 2 mg/m² orally twice daily (approximately every 12 hours) for the first 21 days of each 28-day cycle.
- Maximum dose: 4 mg twice daily
- Continue treatment until disease progression or unacceptable toxicity
- Dosage based on body surface area (BSA):
  - 0.40 to 0.69 m²: 1 mg twice daily
  - 0.70 to 1.04 m²: 2 mg twice daily
  - 1.05 to 1.49 m²: 3 mg twice daily
  - ≥1.50 m²: 4 mg twice daily
Adverse Reactions:
- Adults: Common adverse reactions (≥25%) include rash (90%), diarrhea (59%), nausea (52%), musculoskeletal pain (41%), vomiting (38%), and fatigue (29%).
- Pediatrics: Common adverse reactions (≥25%) include rash (73%), diarrhea (55%), musculoskeletal pain (41%), abdominal pain (39%), vomiting (39%), headache (34%), paronychia (32%), left ventricular dysfunction (27%), and nausea (27%).
Contraindications:None.
Precautions:
- Ocular Toxicity: May cause retinal vein occlusion, retinal pigment epithelium detachment, and blurred vision. Conduct comprehensive ophthalmic assessments prior to initiation and periodically during treatment.
- Cardiac Effects: Monitor left ventricular ejection fraction (LVEF) before and during treatment.
- Dermatologic Reactions: Monitor for rash and manage accordingly.
- Embryo-Fetal Toxicity: Can cause fetal harm. Advise effective contraception during treatment and for 6 weeks (females) or 3 months (males) after the last dose.

Interactions

Drug Interactions: No specific drug interaction studies have been conducted. Use caution when administering with other medications.

Note:

If there is a new packaging for the drug, the new packaging shall prevail. The above information is sourced from DengYue Medicine.
It is only for internal discussion among medical staff and does not serve as a basis for medication. For specific medication guidelines, please consult the attending physician.