GOMEKLI (mirdametinib)

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The first and only FDA-approved treatment for adults with NF1-PN*

*Adults with symptomatic PN not amenable to complete resection.

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Image of an actor portraying an adult patient with NF1-PN

GOMEKLI is proven to shrink plexiform neurofibromas (PNs) in adults with neurofibromatosis type 1 (NF1)1

Primary endpoint: confirmed overall response rate (ORR)1,†

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achieved a confirmed response‡

(≥20% reduction in PN volume on consecutive scans)(95% CI: 29-55)
Of the patients with a confirmed response:
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had a deep response

(>50% reduction in PN volume)2

(This analysis was post hoc and exploratory)

In a post hoc subgroup analysis:

Patients who achieved a deep response had a longer duration of treatment than those who did not3

The median duration of treatment for patients with a deep response was 37 months (range: 20 to 46 months) vs 26 months (range: 7 to 33 months) for those with a 20% to 50% reduction in PN volume.

†Confirmed 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

‡All partial responses.1

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Do you have patients who are starting GOMEKLI?

Download the GOMEKLI Treatment Access Guide for information on how to order GOMEKLI, enroll patients in the CareConnections support program, and more.

Depth of response achieved by adults2,4

Best percent change in PN volume
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Image of a graph showing best tumor volume change from baseline in GOMEKLI treated patients. Please see Prescribing Information.
Image of a graph showing best tumor volume change from baseline in GOMEKLI treated patients. Please see Prescribing Information.
Image of a graph showing best tumor volume change from baseline in GOMEKLI treated patients. Please see Prescribing Information.
Image of a graph showing best tumor volume change from baseline in GOMEKLI treated patients. Please see Prescribing Information.
Data for 50 out of 58 patients shown here; 8 patients did not have postbaseline MRI assessments. Green bars represent those patients who had a confirmed overall response (≥20% reduction in PN volume on consecutive MRI scans). Gray bars represent nonresponders who had a best overall response of stable disease or progressive disease.
Median best change in
PN volume from baseline2:

-41%

(range: −90% to 13%)

Maximum best change in
PN volume from baseline2:

-90%

 Analysis of best change was a prespecified exploratory endpoint.

40-year-old male with a head/neck PN2

Baseline

Image of a 40-year-old male with head/neck PN at baseline in ReNeu case study.
X-ray image of a 40-year-old male with head/neck PN at baseline in ReNeu case study.

Cycle 36

Image of a 40-year-old male with head/neck PN at cycle 36 in ReNeu case study.
X-ray image of a 40-year-old male with a head/neck PN at cycle 36 in ReNeu case study

PN with a volume of 281 mL at baseline was reduced to 69 mL (-79%) at Cycle 36.§

Tumor edges are indicated in cyan. Individual results may vary.

§Long-term follow-up phase of the GOMEKLI ReNeu study.

Durable response in adult patients1,2

88% (21/24) of the confirmed overall responses remained durable for ≥12 months1,ǁ

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Image of a graph showing duration of treatment and response status. Please see Prescribing Information.
Image of a graph showing duration of treatment and response status. Please see Prescribing Information.
Image of a graph showing duration of treatment and response status. Please see Prescribing Information.

Best overall response status among the 58 adult patients taking GOMEKLI2,4

Complete response

0 patients

Partial response#

24 patients

Stable disease**

26 patients

Progressive disease§§

0 patients

Not evaluable††

8 patients

  • All evaluable patients had either an objective response or stable disease based on best overall confirmed response
  • 6 patients had disease progression during the treatment phase
Stable disease and progressive disease were exploratory endpoints and data should be interpreted with caution as ReNeu is a single-arm trial.
  • 50% (12/24) of the confirmed overall responses remained durable for ≥24 months1,ǁ
  • 46% of responders (11/24) had onset of response at Cycle 5, the first on-treatment assessment (based on a post hoc analysis)4
  • Median time to first confirmed response was 7.8 months (range: 4 to 19 months)1

84% of patients

(26/31) who completed the treatment phase chose to remain on GOMEKLI for long-term follow-up.2

 ǁDuration of response was assessed based on observed time.1

Four-week cycles of 3 weeks on/1 week off. Treatment phase ends 3 weeks into final cycle.2

#Partial response corresponds with confirmed overall response of ≥20% reduction in target PN volume from baseline.1

**Stable disease is defined as a <20% increase or a <20% decrease in target PN volume from baseline.2

††Patients who were not evaluable were those for whom no postbaseline volumetric data were collected.4

‡‡Responses were confirmed on a subsequent scan within 2 to 6 months.2

§§Progressive disease is defined as a ≥20% increase in target PN volume from baseline.2

ǁǁData cutoff was September 20, 2023.2

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Discussing GOMEKLI with your patients

The GOMEKLI Patient Brochure can make the ReNeu study data more approachable and easier for you to explain to patients.

Download brochure
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Want to learn more about GOMEKLI treatment?

The GOMEKLI Overview Brochure provides an overview of GOMEKLI clinical data for both adult and pediatric patients with NF1-PN.
Download guide
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GOMEKLI safety profile in adults

The majority of adverse reactions were mild to moderate and manageable.1

Review the data

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. Published online November 8, 2024. doi.org/10.1200/JCO.24.01034
  3. Gershon T, Moertel C, McNall-Knapp RY, et al. Pivotal, phase 2b ReNeu trial of mirdametinib in children and adults with neurofibromatosis type 1-associated plexiform neurofibroma (NF1-PN): a spotlight on patients achieving deep response. Poster presented at: 29th Annual Meeting of the Society for Neuro-Oncology; November 21-24, 2024; Houston, TX.
  4. Data on file: SpringWorks Therapeutics, Inc.