GOMEKLI (mirdametinib)

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Frequently asked questions about GOMEKLI (mirdametinib)

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About GOMEKLI

Yes, GOMEKLI is the first FDA-approved treatment for both adults and children 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection.1

The efficacy and safety of GOMEKLI were evaluated in both adult and pediatric patients in the phase 2b, single-arm ReNeu study.1,2 See the ReNeu study design.

Please see Important Safety Information and full Prescribing Information.

GOMEKLI is a selective and targeted systemic therapy that works by inhibiting mitogen-activated protein kinase kinases 1 and 2 (MEK1/2), key mediators in the mitogen-activated protein kinase (MAPK) pathway.1-3

In NF1-PN, an overactive MAPK pathway can lead to excessive cell growth and tumor formation. In preclinical models, GOMEKLI helped to downregulate the MAPK pathway, reducing the proliferation and survival of tumor cells.1,4,5,6 Explore the mechanism of action of GOMEKLI.

Efficacy

The primary endpoint was confirmed overall response rate (ORR) defined as the proportion of patients with complete response (disappearance of the target PN) or partial response (≥20% reduction) on magnetic resonance imaging (MRI) of the target PN volume from baseline to Cycle 24 (treatment phase) as assessed by blinded independent central review (BICR) on ≥2 consecutive scans within 2 to 6 months.1 Review the results in adults and the results in children.

Secondary endpoints included duration of response; change in patient-reported outcomes, including worst tumor pain severity, pain interference, and health-related quality of life, from baseline to Cycle 13; and safety and tolerability.1,2

Please see Important Safety Information and full Prescribing Information.

In the adult cohort of ReNeu, the median time to onset of confirmed response was 7.8 months (range: 4 to 19 months). A post hoc analysis showed that 46% of responders (11/24) had onset of response at Cycle 5, the first on-treatment assessment.1,7 See the onset and duration of response in adults.

Similarly, in the pediatric cohort, the median time to onset of confirmed response was 7.9 months (range: 4.1 to 18.8 months); 45% of responders (13/29) had onset of response at Cycle 5, the first on-treatment assessment (based on a post hoc analysis).1,7 See the onset and duration of response in children.

Please see Important Safety Information and full Prescribing Information.

Yes, the latest findings from the exploratory long-term follow-up analysis from ReNeu show that longer duration of GOMEKLI treatment improved confirmed ORR, with some deep responses achieved. Additionally, no new safety signals emerged in either cohort.8

The median duration of GOMEKLI treatment was 21.8 months (range: 0.4 to 54.4 months) in adult patients and 25.4 months (range: 1.6 to 48.5 months) in pediatric patients.8 See the long-term data in adults and long-term data in children.

The exploratory long-term follow-up analysis from ReNeu is not included in the full Prescribing Information of GOMEKLI.

Safety

GOMEKLI has no contraindications.1

The GOMEKLI Prescribing Information includes Warning and Precautions on ocular toxicity, left ventricular dysfunction, dermatologic adverse reactions, and embryo-fetal toxicity.

Please see Important Safety Information and full Prescribing Information.

In adults, the most common adverse reactions (>25%) were rash, diarrhea, nausea, musculoskeletal pain, vomiting, and fatigue.1 See the safety data in adults.

In children, the most common adverse reactions (>25%) were rash, diarrhea, musculoskeletal pain, abdominal pain, vomiting, headache, paronychia, left ventricular dysfunction, and nausea.1 See the safety data in children.

Please see Important Safety Information and full Prescribing Information.

Dosing and administration

The recommended dosage for GOMEKLI is 2 mg/m2 orally twice daily (approximately every 12 hours), with or without food, for the first 21 days of each 28-day cycle. The maximum dose is 4 mg twice daily.1

The recommended dose of GOMEKLI is based on body surface area.1 Review the recommended dosing.

Yes, GOMEKLI is available in 2 dosing formulations: a capsule that comes in 2 sizes, and a tablet for oral suspension that can either be swallowed whole or dispersed in water for those patients who have difficulty swallowing.1 See how to help patients prepare GOMEKLI for oral suspension.

Support for your patients and practice

SpringWorks CareConnections® provides personalized support services and resources to help patients get started and stay on track with GOMEKLI, including coverage and access support, financial assistance, and educational and emotional support. Learn more about the SpringWorks CareConnections patient support program.

Additionally, the GOMEKLI patient website has a variety of downloadable tools and videos designed to support patients throughout their treatment journey.

GOMEKLI is exclusively available through a limited specialty pharmacy network.

GOMEKLI is available to order through eligible medically integrated dispensing pharmacies by ordering directly from one of our specialty distributor partners. You can download the GOMEKLI Product Fact Sheet and Ordering Guide for additional information.

Additional questions?

Reach out to a SpringWorks representative to stay connected and learn more about GOMEKLI.

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Indication

GOMEKLI (mirdametinib) is indicated for the treatment of adult and pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection.

Important Safety Information

Warnings and Precautions

Ocular Toxicity: GOMEKLI can cause ocular toxicity including retinal vein occlusion (RVO), retinal pigment epithelium detachment (RPED), and blurred vision. In the adult pooled safety population, ocular toxicity occurred in 28% of patients treated with GOMEKLI: 21% were Grade 1, 5% were Grade 2 and 1.3% were Grade 3. RVO occurred in 2.7%, RPED occurred in 1.3%, and blurred vision occurred in 9% of adult patients. In the pediatric pooled safety population, ocular toxicity occurred in 19% of patients: 17% were Grade 1 and 1.7% were Grade 2. Conduct comprehensive ophthalmic assessments prior to initiating GOMEKLI, at regular intervals during treatment, and to evaluate any new or worsening visual changes such as blurred vision. Continue, withhold, reduce the dose, or permanently discontinue GOMEKLI as clinically indicated.

Left Ventricular Dysfunction: GOMEKLI can cause left ventricular dysfunction. GOMEKLI has not been studied in patients with a history of clinically significant cardiac disease or LVEF <55% prior to initiation of treatment. In the ReNeu study, decreased LVEF of 10 to <20% occurred in 16% of adult patients treated with GOMEKLI. Five patients (9%) required dose interruption, one patient (1.7%) required a dose reduction, and one patient required permanent discontinuation of GOMEKLI. The median time to first onset of decreased LVEF in adult patients was 70 days. Decreased LVEF of 10 to <20% occurred in 25%, and decreased LVEF of ≥20% occurred in 1.8% of pediatric patients treated with GOMEKLI. One patient (1.8%) required dose interruption of GOMEKLI. The median time to first onset of decreased LVEF in pediatric patients was 132 days. All patients with decreased LVEF were identified during routine echocardiography, and decreased LVEF resolved in 75% of patients. Before initiating GOMEKLI, assess ejection fraction (EF) by echocardiogram. Monitor EF every 3 months during the first year and then as clinically indicated. Withhold, reduce the dose, or permanently discontinue GOMEKLI based on severity of adverse reaction.

Dermatologic Adverse Reactions: GOMEKLI can cause dermatologic adverse reactions including rash. The most frequent rashes included dermatitis acneiform, rash, eczema, maculo-papular rash and pustular rash. In the pooled adult safety population, rash occurred in 92% of patients treated with GOMEKLI (37% were Grade 2 and 8% were Grade 3) and resulted in permanent discontinuation in 11% of patients. In the pooled pediatric safety population, rash occurred in 72% of patients treated with GOMEKLI (22% were Grade 2 and 3.4% were Grade 3) and resulted in permanent discontinuation in 3.4% of patients. Initiate supportive care at first signs of dermatologic adverse reactions. Withhold, reduce the dose, or permanently discontinue GOMEKLI based on severity of adverse reaction.

Embryo-Fetal Toxicity: GOMEKLI can cause fetal harm when administered to a pregnant woman. Verify the pregnancy status of females of reproductive potential prior to the initiation of GOMEKLI. Advise pregnant women and females of reproductive potential of the potential risk to a fetus. Also advise patients to use effective contraception during treatment with GOMEKLI and for 6 weeks after the last dose (females) or 3 months after the last dose (males).

Adverse Reactions

The most common adverse reactions (>25%) in adult patients were rash (90%), diarrhea (59%), nausea (52%), musculoskeletal pain (41%), vomiting (38%), and fatigue (29%). Serious adverse reactions occurred in 17% of adult patients who received GOMEKLI. The most common Grade 3 or 4 laboratory abnormality (>2%) was increased creatine phosphokinase.

The most common adverse reactions (>25%) in pediatric patients were rash (73%), diarrhea (55%), musculoskeletal pain (41%), abdominal pain (39%), vomiting (39%), headache (34%), paronychia (32%), left ventricular dysfunction (27%), and nausea (27%). Serious adverse reactions occurred in 14% of pediatric patients who received GOMEKLI. The most common Grade 3 or 4 laboratory abnormalities (>2%) were decreased neutrophil count and increased creatine phosphokinase.

Use in Specific Populations

Verify the pregnancy status of patients of reproductive potential prior to initiating GOMEKLI. Due to the potential for adverse reactions in a breastfed child, advise patients not to breastfeed during treatment with GOMEKLI and for 1 week after the last dose.

Indication

GOMEKLI (mirdametinib) is indicated for the treatment of adult and pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection.
To report SUSPECTED ADVERSE REACTIONS, contact SpringWorks Therapeutics Inc. at 1-888-400-7989 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Please click here for full Prescribing Information, including Patient Information and Instructions for Use.
  1. GOMEKLI. Prescribing Information. SpringWorks Therapeutics, Inc.
  2. Moertel CL, Hirbe AC, Shuhaiber HH, et al. ReNeu: a pivotal, phase IIb trial of mirdametinib in adults and children with symptomatic neurofibromatosis type 1-associated plexiform neurofibroma. J Clin Oncol. 2025;43(6):716-729.
  3. Cheng Y, Tian H. Current development status of MEK inhibitors. Molecules. 2017;22(10):1551.
  4. Gutmann DH, Parada LF, Silva AJ, Ratner N. Neurofibromatosis type 1: modeling CNS dysfunction. J Neurosci. 2012;32(41):14087-14093.
  5. Gutmann DH, Ferner RE, Listernick RH, Korf BR, Wolters PL, Johnson KJ. Neurofibromatosis type 1. Nat Rev Dis Primers. 2017;3:17004.
  6. Wang D, Boerner SA, Winkler JD, LoRusso PM. Clinical experience of MEK inhibitors in cancer therapy. Biochim Biophys Acta. 2007;1773(8):1248-1255.
  7. Data on file: SpringWorks Therapeutics, Inc.
  8. Hirbe AC, Moertel CL, Shuhaiber HH, et al. Update from the long-term follow-up (LTFU) phase of ReNeu: a pivotal phase 2b trial of mirdametinib in children and adults with neurofibromatosis type 1 (NF1)-associated symptomatic plexiform neurofibroma (PN). Presented at: 2025 Global NF Conference; June 21-24, 2025; Washington, DC.