2 days ago · Gomekli (mirdametinib) is an FDA-approved medication used to treat neurofibromatosis type 1 (NF1) with plexiform neurofibromas causing symptoms that cannot be surgically removed. Gomekli helps to reduce tumor size, relieve symptoms, and improve quality of life. Gomekli can be used by adults and patients 2 years and older.

On February 11, 2025, the Food and Drug Administration approved mirdametinib (Gomekli, SpringWorks Therapeutics, Inc.), a kinase inhibitor, for adult and pediatric patients 2 years of age and...

Feb 11, 2025 · The FDA approval of GOMEKLI is based on results from the Phase 2b ReNeu trial, which enrolled 114 patients with NF1-PN ≥2 years of age (58 adults and 56 pediatric patients). 10 GOMEKLI met the primary endpoint of confirmed objective response rate (ORR), as assessed by blinded independent central review, demonstrating a 41% ORR (N= 24/ 58) in ...

1 day ago · Gomekli is the first treatment of its kind to be approved for both children and adults with the condition. The drug will be available in 1 mg and 2 mg capsules or a 1 mg tablet for oral suspension.

3 days ago · Mirdametinib, sold under the brand name Gomekli, is a medication used for the treatment of people with neurofibromatosis type 1. [1] Mirdametinib is a kinase inhibitor. [1] [2] It is taken by mouth.[1]The most common adverse reactions in adults include rash, diarrhea, nausea, musculoskeletal pain, vomiting, and fatigue. [3] The most common grade 3 or 4 laboratory …

Feb 12, 2025 · The FDA approval of GOMEKLI is based on results from the Phase 2b ReNeu trial, which enrolled 114 patients with NF1-PN ≥2 years of age (58 adults and 56 pediatric patients). 10 GOMEKLI met the primary endpoint of confirmed objective response rate (ORR), as assessed by blinded independent central review, demonstrating a 41% ORR (N= 24/ 58) in ...

6 days ago · Both Gomekli and Koselugo are kinase inhibitors that target mitogen-activated protein kinase kinases 1 and 2. These enzymes are responsible for cell growth and are overactive in patients with NF1. SpringWorks’ drug demonstrated objective response rates of tumour volume reduction in 41% and 52% of adults and children respectively in the Phase ...

On February 11, 2025, the U.S. Food and Drug Administration approved mirdametinib (Gomekli ™), a kinase inhibitor, for adult and pediatric patients aged 2 and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) …

Feb 12, 2025 · The FDA approval of GOMEKLI is based on results from the Phase 2b ReNeu trial, which enrolled 114 patients with NF1-PN ≥2 years of age (58 adults and 56 pediatric patients). 10 GOMEKLI met the ...

Feb 12, 2025 · Gomekli demonstrated an ORR of 41% in adults and 52% in children, with durable tumor reductions and improvements in pain and quality of life. Additionally, 88% of adults and 90% of children with a confirmed response reported a response that lasted at least one full year. Further, 50% of adults and 48% of children had a response of at least two ...