What Is Gomekli? Uses, Dosage, and What You Need to Know in 2026

Gomekli (mirdametinib) is a relatively new prescription medication that represents a major advancement for people living with neurofibromatosis type 1 (NF1). Approved by the FDA in February 2025, it is the first treatment specifically approved for both adults and children with NF1-related plexiform neurofibromas — tumors that grow along nerves and often cannot be surgically removed. This guide covers the basics: what Gomekli is, what it treats, how it's taken, and what the research shows.

What Is Gomekli Used For?

Gomekli's FDA-approved indication is for adults and children aged 2 years and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) that cannot be completely removed by surgery.

Neurofibromatosis Type 1 (NF1) is a rare genetic disorder caused by mutations in the NF1 gene on chromosome 17. The NF1 gene normally produces a protein called neurofibromin that helps regulate cell growth. When the NF1 gene is mutated, neurofibromin is deficient, leading to uncontrolled cell growth and the formation of tumors — including plexiform neurofibromas (PN).

Plexiform neurofibromas grow along peripheral nerve sheaths and often infiltrate into surrounding tissues. They can cause significant pain, disfigurement, and loss of function. Because of their infiltrative nature, many PNs cannot be completely surgically removed without causing major complications — which is why medications like Gomekli are needed.

Who Is Gomekli For?

Gomekli is approved for:

Adults (18 years and older) with NF1 who have symptomatic, inoperable plexiform neurofibromas

Pediatric patients aged 2 years and older with NF1 who have symptomatic, inoperable plexiform neurofibromas

Gomekli is not currently approved for other types of NF1 tumors such as cutaneous neurofibromas, malignant peripheral nerve sheath tumors (MPNSTs), or NF2 (neurofibromatosis type 2).

How Is Gomekli Taken?

Gomekli is taken orally (by mouth) in a distinctive dosing schedule:

Dose: 2 mg/m² twice daily (approximately 12 hours apart), with or without food

Schedule: 21 days on, followed by 7 days off (28-day cycle)

Maximum dose: 4 mg twice daily

Duration: Continue until disease progression or unacceptable side effects

Gomekli comes in two forms:

Capsules: 1 mg and 2 mg strengths. Swallow whole — do not open, break, or chew.

Tablets for oral suspension: 1 mg grape-flavored dispersible tablets. Can be swallowed whole or dissolved in water. Especially useful for young children who have difficulty swallowing capsules.

What Does the Research Show?

The FDA approved Gomekli based on results from the Phase 2b ReNeu clinical trial (NCT03962543), which enrolled 114 patients (58 adults and 56 children) with symptomatic, inoperable NF1-associated PNs. Key results:

Overall response rate of 41% in adults (24 of 58 patients had their tumor shrink by 20% or more)

Overall response rate of 52% in children (29 of 56 patients had confirmed tumor reduction)

Median best tumor volume reduction of -41% in adults and -42% in children

88% of adult responders and 90% of pediatric responders maintained their response for at least 12 months

Patients also reported less pain and better quality of life from early in treatment through Cycle 13

What Drug Class Is Gomekli?

Gomekli belongs to the drug class known as MEK inhibitors (mitogen-activated protein kinase kinase inhibitors). It works by blocking the MEK1 and MEK2 proteins, which are part of the RAS-MAPK signaling pathway. This pathway is abnormally overactive in NF1, driving tumor growth. By blocking MEK1/2, Gomekli interrupts this signal and slows or reverses neurofibroma growth.

Is There a Generic Version of Gomekli?

No. As of 2026, Gomekli (mirdametinib) is only available as the brand-name product from SpringWorks Therapeutics. There is no generic version of mirdametinib. As a newly approved drug treating a rare condition, generic availability is many years away. Financial assistance programs are available to help manage costs — see our guide on

Where Can I Get Gomekli?

Gomekli is only dispensed through specialty pharmacies. Your prescribing specialist will coordinate the referral to a specialty pharmacy. If you need help identifying which specialty pharmacies near you can fill your prescription, medfinder calls pharmacies on your behalf and texts you the results.

For a deeper explanation of how Gomekli works at the molecular level, see our article on how Gomekli works — mechanism of action explained.