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# SpringWorks’ Gomekli Approved for Neurofibromatosis
- URL: https://www.fdaweb.com/springworks-gomekli-approved-for-neurofibromatosis/
- Published: 2025-02-11T12:00:00.000Z
- Updated: 2026-09-14T14:52:53.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5158643

FDA has approved a SpringWorks Therapeutics NDA for Gomekli (mirdametinib), a kinase inhibitor for treating adult and pediatric patients two years of age and older with neurofibromatosis Type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection.

Approval was based on data from the ReNeu (NCT03962543) multicenter, single-arm trial in 114 patients with symptomatic, inoperable NF1-associated PN causing significant morbidity, an FDA [release says](https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-mirdametinib-adult-and-pediatric-patients-neurofibromatosis-type-1-who-have-symptomatic?ref=fdaweb.com). “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 (greater than 20% reduction in PN volume).”

Regarding safety, the most common adverse reactions (greater than 25%) in adult patients were rash, diarrhea, nausea, musculoskeletal pain, vomiting, and fatigue, FDA says. The most common Grade 3 or 4 laboratory abnormality was increased creatine phosphokinase.

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