Initial US Approval:
2025
Key Clinical Studies:
ReNeu (NCT03962543)
Drug Class/Description:
Kinase inhibitor
Indications and Usage:
- Indicated for the treatment of adult and pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection.
Dosage Administration:
- The recommended dosage of Gomekli is 2 mg/m² orally twice daily, with or without food, for the first 21 days of each 28-day cycle. Continue treatment with Gomekli until disease progression or unacceptable toxicity.
Dosage Forms and Strengths:
- Capsules: 1 mg and 2 mg.
- Tablets for Oral Suspension: 1 mg.
Contraindications:
None.
Warnings and Precautions:
- Ocular Toxicity: Conduct comprehensive ophthalmic assessments prior to initiating Gomekli, at regular intervals during treatment, and for new or worsening visual changes or blurred vision. Continue, withhold, reduce the dose, or permanently discontinue Gomekli based on severity.
- Left Ventricular Dysfunction: Assess ejection fraction by echocardiogram prior to initiating Gomekli, every 3 months during the first year, then as clinically indicated thereafter. Withhold, reduce the dose, or permanently discontinue Gomekli based on severity.
- Dermatologic Adverse Reactions: Initiate supportive care at first signs of dermatologic adverse reactions including rash. Withhold, reduce the dose, or permanently discontinue Gomekli based on severity.
- Embryo-Fetal Toxicity: Can cause fetal harm. Advise patients of reproductive potential of the potential risk to a fetus and to use effective contraception.
Adverse Reactions:
Adults:
- The most common adverse reactions (> 25%) were rash, diarrhea, nausea, musculoskeletal pain, vomiting, and fatigue.
- The most common Grade 3 or 4 laboratory abnormality (> 2%) was increased creatine phosphokinase.
Pediatric patients:
- The most common adverse reactions (> 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 (> 2%) were decreased neutrophil count and increased creatine phosphokinase.
Use in Specific Populations:
- Lactation: Advise not to breastfeed.
- Infertility: May impair fertility in females.
Adapted from:
https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/219379Orig1s000lbl.pdf
Every health-care provider should make their own determination regarding specific safe and appropriate patient care practices, including drug dosages and indications. The provider should always consult the most recent prescribing/product information. FDA Focus information is not guaranteed to be accurate, complete, or current. JADPRO and its editors, authors, reviewers, and commentators cannot be held responsible for any liability incurred as a consequence of the application of any of the information listed within.
