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No clinically important drug-drug interactions have been observed with GOMEKLI.1 Learn more.
In in vitro studies:
In a model-informed approach:
ERK=extracellular signal-regulated kinase; MAPK=mitogen-activated protein kinase; MEK=mitogen-activated protein kinase kinase; RAF=rapidly accelerated fibrosarcoma; RAS=rat sarcoma viral oncogene homolog.
Patients had to be ≥2 years of age and have NF1 with a symptomatic, inoperable PN.1,†
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 months1
*Patients were required to return for a safety follow-up 30 days after their last dose of study treatment.6
†Patients were excluded if they had lymphoma, leukemia, or any malignancy (including malignant glioma or MPNST) within the past 5 years; breast cancer within the past 10 years; or evidence of an active optic glioma or other low-grade glioma requiring treatment with chemotherapy or radiation therapy.6
Adult
cohort
(n=56)
Median age at enrollment (range)
34 years (18-69)
Sex
Male
36%
46%
Female
64%
Target PN progressing at study entry
53%
62%
Location of target PN
48%
50%
Lower extremities
26%
7%
Paraspinal
9%
7%
Chest wall
7%
4%
Mesentery and pelvis
2%
9%
Upper extremities
3%
7%
Abdominal wall
0%
2%
Other
5%
14%
Type of PN-related morbidity‡
Pain
90%
70%
Disfigurement or major deformity
52%
50%
Motor dysfunction
40%
27%
Airway dysfunction
5%
12%
Other
17%
21%
Previous PN treatment
Surgery
69%
36%
19%
14%
Radiotherapy
2%
0%
‡Morbidities were clinician determined. Patients may have had more than 1 reported morbidity.
GOMEKLI was proven effective in both adults and children living with NF1-PN.1
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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