Top Qs
Timeline
Chat
Perspective

Mirdametinib

Medication From Wikipedia, the free encyclopedia

Mirdametinib
Remove ads

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]

Quick Facts Clinical data, Trade names ...

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 abnormalities include increased creatine phosphokinase.[3] The most common adverse reactions in children include rash, diarrhea, musculoskeletal pain, abdominal pain, vomiting, headache, paronychia, left ventricular dysfunction, and nausea.[3] The most common grade 3 or 4 laboratory abnormalities include decreased neutrophil count and increased creatine phosphokinase.[3]

Mirdametinib was approved for medical use in the United States in February 2025.[1][3]

Remove ads

Medical uses

Mirdametinib is indicated for the treatment of people with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas not amenable to complete resection.[1]

Adverse effects

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 abnormalities include increased creatine phosphokinase.[3] The most common adverse reactions in children include rash, diarrhea, musculoskeletal pain, abdominal pain, vomiting, headache, paronychia, left ventricular dysfunction, and nausea.[3] The most common grade 3 or 4 laboratory abnormalities include decreased neutrophil count and increased creatine phosphokinase.[3]

Mirdametinib can cause left ventricular dysfunction and ocular toxicity including retinal vein occlusion, retinal pigment epithelial detachment, and blurred vision.[3]

Remove ads

History

The efficacy of mirdametinib was evaluated in ReNeu (NCT03962543), a multicenter, single-arm trial in 114 participants aged two years of age and older (58 adults, 56 pediatric participants) with symptomatic, inoperable NF1-associated plexiform neurofibromas causing significant morbidity.[3] An inoperable plexiform neurofibromas was defined as a plexiform neurofibromas that could not be completely surgically removed without risk for substantial morbidity due to encasement or close proximity to vital structures, invasiveness, or high vascularity.[3]

The US Food and Drug Administration (FDA) granted the application for mirdametinib priority review, fast track, and orphan drug designations along with a priority review voucher.[3]

Society and culture

Mirdametinib was approved for medical use in the United States in February 2025.[3][4][5]

In May 2025, the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion, recommending the granting of a conditional marketing authorization for the medicinal product Ezmekly, intended for the treatment of plexiform neurofibromas (PN) in adults and children from two years of age with neurofibromatosis type 1 (NF1).[6] The applicant for this medicinal product is SpringWorks Therapeutics Ireland Limited.[6]

Remove ads

References

Loading related searches...

Wikiwand - on

Seamless Wikipedia browsing. On steroids.

Remove ads