Mirdametinib, sold under the brand name Gomekli, is a

medication Medication (also called medicament, medicine, pharmaceutical drug, medicinal product, medicinal drug or simply drug) is a drug used to medical diagnosis, diagnose, cure, treat, or preventive medicine, prevent disease. Drug therapy (pharmaco ...

used for the treatment of people with

neurofibromatosis type 1 Neurofibromatosis (NF) refers to a group of three distinct genetic conditions in which tumors grow in the nervous system. The tumors are non-cancerous (benign) and often involve the skin or surrounding bone. Although symptoms are often mild, e ...

. Mirdametinib is a kinase inhibitor. It is taken

by mouth Oral administration is a route of administration whereby a substance is taken through the Human mouth, mouth, swallowed, and then processed via the digestive system. This is a common route of administration for many medications. Oral administ ...

. The most common adverse reactions in adults include rash, diarrhea, nausea, musculoskeletal pain, vomiting, and fatigue. The most common grade 3 or 4 laboratory abnormalities include increased

creatine phosphokinase Creatine kinase (CK), also known as creatine phosphokinase (CPK) or phosphocreatine kinase, is an enzyme () expressed by various tissues and cell types. CK catalyses the conversion of creatine and uses adenosine triphosphate (ATP) to create phos ...

. The most common adverse reactions in children include rash, diarrhea, musculoskeletal pain, abdominal pain, vomiting, headache,

paronychia Paronychia is an inflammation of the skin around the nail, often due to bacteria or fungi. Its sudden (acute) occurrence is usually due to the bacterium ''Staphylococcus aureus''. Gradual (chronic) occurrences are typically caused by fungi ...

left ventricular dysfunction Left may refer to: Music * ''Left'' (Hope of the States album), 2006 * ''Left'' (Monkey House album), 2016 * ''Left'' (Helmet album), 2023 * "Left", a song by Nickelback from the album ''Curb'', 1996 Direction * Left (direction), the relativ ...

, and nausea. The most common grade 3 or 4 laboratory abnormalities include decreased

neutrophil Neutrophils are a type of phagocytic white blood cell and part of innate immunity. More specifically, they form the most abundant type of granulocytes and make up 40% to 70% of all white blood cells in humans. Their functions vary in differe ...

count and increased creatine phosphokinase. Mirdametinib was approved for medical use in the United States in February 2025.

Medical uses

Mirdametinib is

indicated In medicine, an indication is a valid reason to use a certain test, medication, procedure, or surgery. There can be multiple indications to use a procedure or medication. An indication can commonly be confused with the term diagnosis. A diagnosis ...

for the treatment of people with neurofibromatosis type 1 who have symptomatic

plexiform neurofibroma A neurofibroma is a benign nerve-sheath tumor in the peripheral nervous system. In 90% of cases, they are found as stand-alone tumors (solitary neurofibroma, solitary nerve sheath tumor or sporadic neurofibroma), while the remainder are found in ...

s not amenable to complete resection.

Adverse effects

The most common adverse reactions in adults include rash, diarrhea, nausea, musculoskeletal pain, vomiting, and fatigue. The most common grade 3 or 4 laboratory abnormalities include increased creatine phosphokinase. The most common adverse reactions in children include rash, diarrhea, musculoskeletal pain, abdominal pain, vomiting, headache, paronychia, left ventricular dysfunction, and nausea. The most common grade 3 or 4 laboratory abnormalities include decreased neutrophil count and increased creatine phosphokinase. Mirdametinib can cause left ventricular dysfunction and ocular toxicity including

retinal vein occlusion Central retinal vein occlusion, also CRVO, is when the central retinal vein becomes occluded, usually through thrombosis. The central retinal vein is the venous equivalent of the central retinal artery and both may become occluded. Since the cent ...

, retinal pigment epithelial detachment, and blurred vision.

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. 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. The US

Food and Drug Administration The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is respo ...

(FDA) granted the application for mirdametinib

priority review Priority review is a program of the United States Food and Drug Administration (FDA) to expedite the review process for drugs that are expected to have a particularly great impact on the treatment of a disease. The priority review voucher program ...

fast track Fast Track, Fast track, or Fasttrack may refer to: Processes and systems * Fast track (FDA), a U.S. Food and Drug Administration expedited review program * Fast track (trade), the authority of the U.S. President to broker trade agreements with lim ...

, and

orphan drug An orphan drug is a medication, pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by th ...

designations along with a priority review voucher.

Society and culture

Legal status

Mirdametinib was approved for medical use in the United States in February 2025. In May 2025, the

Committee for Medicinal Products for Human Use The Committee for Medicinal Products for Human Use (CHMP), formerly known as the Committee for Proprietary Medicinal Products (CPMP), is the European Medicines Agency's committee responsible for elaborating the agency's opinions on all issues regar ...

of the

European Medicines Agency The European Medicines Agency (EMA) is an agency of the European Union (EU) in charge of the evaluation and supervision of pharmaceutical products. Prior to 2004, it was known as the European Agency for the Evaluation of Medicinal Products ...

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). The applicant for this medicinal product is SpringWorks Therapeutics Ireland Limited. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.

References

External links

* * {{Authority control MEK inhibitors Orphan drugs Tyrosine kinase inhibitors Fluorobenzene derivatives Iodobenzene derivatives Anilines Benzamides Glycerol ethers