Gomekli Approved for Patients With Neurofibromatosis Type 1

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

Efficacy was evaluated in ReNeu (ClinicalTrials.gov Identifier: NCT03962543), a multicenter, single-arm trial in 114 patients 2 years of age and older (58 adults, 56 pediatric patients) with symptomatic, inoperable