FDA

Gomekli OK’d for neurofibromatosis type 1-related neurofibromas

February 13, 2025

Brand name: Gomekli

Generic name: mirdametinib

Manufacturer: SpringWorks Therapeutics

Approval date: February 11, 2025

FDA approved Gomekli (mirdametinib), a kinase inhibitor, for adult and pediatric patients ≥2 years of age with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection.

Efficacy

Approval was based on data from the phase 2b ReNeu trial (NCT03962543) involving 114 patients ≥2 years of age (58 adults, 56 pediatric patients) with symptomatic, inoperable NF1-associated PN causing significant morbidity.

The major efficacy outcome measure was confirmed overall response rate (ORR), defined as the percentage of patients with complete response (disappearance of the target PN) or partial response (≥20% reduction in PN volume). Confirmed ORR was 41% for adults (95% confidence interval [CI], 29-55) and 52% in the pediatric cohort (95% CI, 38-65).

Safety

The most common adverse reactions (>25%) in adult patients were rash, diarrhea, nausea, musculoskeletal pain, vomiting, and fatigue. The most common Grade 3 or 4 lab abnormality (>2%) was increased CPK.

The most common adverse reactions (>25%) in pediatric patients were rash, diarrhea, musculoskeletal pain, abdominal pain, vomiting, headache, paronychia, left ventricular dysfunction, and nausea. The most common Grade 3 or 4 lab abnormalities (>2%) were decreased neutrophil count and increased CPK.

Mirdametinib can also cause left ventricular dysfunction and ocular toxicity including retinal vein occlusion, retinal pigment epithelial detachment, and blurred vision. Mirdametinib should be withheld, dosage reduced, or permanently discontinued based on the severity of adverse reactions.

Recommended dose

The recommended dosage of Gomekli is 2 mg/m² body surface area PO bid, with or without food, for the first 21 days of each 28-day cycle. Treatment should be continued until disease progression or unacceptable toxicity.

Sources:

FDA approves mirdametinib for adult and pediatric patients with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas not amenable to complete resection. Food and Drug Administration. 2025. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-mirdametinib-adult-and-pediatric-patients-neurofibromatosis-type-1-who-have-symptomatic

SpringWorks Therapeutics announces FDA approval of Gomekli™ (mirdametinib) for the treatment of adult and pediatric patients with NF1-PN. SpringWorks Therapeutics. 2025. https://ir.springworkstx.com/news-releases/news-release-details/springworks-therapeutics-announces-fda-approval-gomeklitm

Gomekli. Package insert. SpringWorks Therapeutics. https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/219379Orig1s000lbl.pdf