*Children ≥2 years of age with symptomatic PN not amenable to complete resection.
Actor portrayal.
(≥20% reduction in PN volume on consecutive scans)(95% CI: 38-65)
Of the patients with a confirmed response:
(>50% reduction in PN volume)2
(This analysis was post hoc and exploratory)
In a post hoc subgroup analysis:
The median duration of treatment for patients with a deep response was 27 months (range: 22 to 39 months) vs 25 months (range: 12 to 40 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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Data for 54 out of 56 patients shown here; 2 patients did not have postbaseline MRI assessments. Gold 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.
-42%
(range: −91% to 48%)
-91%
View 2 case studies from ReNeu
Baseline
Cycle 21
PN with a volume of 95 mL at baseline was reduced to 48 mL (-49%) at Cycle 21.
Baseline
Cycle 24
PN with a volume of 221 mL at baseline was reduced to 39 mL (-82%) at Cycle 24.
Tumor edges are indicated in cyan. Individual results may vary.
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View additional response data
Complete response
0 patients
29 patients
22 patients
Progressive disease**
3 patients
Not evaluable††
2 patients
Stable disease and progressive disease were exploratory endpoints and data should be interpreted with caution as ReNeu is a single-arm trial.
who completed the treatment phase chose to remain on GOMEKLI for long-term follow-up.2
§Duration of response was assessed based on observed time.1
ǁFour-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
§§Data cutoff was September 20, 2023.2
The GOMEKLI Patient Brochure can make the ReNeu study data more approachable and easier for you to explain to patients.
The GOMEKLI Overview Brochure provides an overview of GOMEKLI clinical data for both adult and pediatric patients with NF1-PN.
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.
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).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.You are now leaving GOMEKLI.com, a website provided by SpringWorks Therapeutics. This link will take you to a different site to which this Privacy Policy and Terms of Use do not apply.
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