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GOMEKLI is a prescription medicine used to treat adults and children 2 years of age and older with neurofibromatosis type 1 (NF1) who have plexiform neurofibromas (PN) that cause symptoms and cannot be completely removed by surgery.
It is not known if GOMEKLI is safe and effective in children younger than 2 years of age.
GOMEKLI is a MEK inhibitor, which is targeted therapy – not traditional chemotherapy. It works by helping to block certain signals in the body that cause plexiform neurofibromas to grow.
GOMEKLI was studied in one of the largest (N=114) clinical trials for neurofibromatosis type 1 with plexiform neurofibromas (NF1-PN).
SAW THEIR PLEXIFORM SHRINK by at least 20%*
SAW THEIR PLEXIFORM SHRINK
by more than 50%†
Plexiform before starting GOMEKLI
20%
SMALLER
50%
SMALLER
*Reduction in plexiform size had to be confirmed on a later magnetic resonance imaging (MRI) scan.
†The 50% reduction in plexiform size was an exploratory analysis, meaning it was not specifically defined in advance of the trial.
The 20% change in plexiform volume is the standard benchmark used in NF1-PN trials.
– Austin, GOMEKLI Patient Ambassador
Individual results may vary.
In adults who benefited from treatment, plexiforms began to shrink by at least 20% within 4 and 19 months of starting GOMEKLI. The median (middle) amount of time it took for plexiforms to begin shrinking was 7.8 months.
84% of adults chose to keep taking GOMEKLI in an optional long-term follow-up phase of the study.
In adults, the most common ones were:
The most common severe abnormal blood test result in adults involved an increased enzyme called creatine phosphokinase (CPK).
GOMEKLI may cause fertility problems in females, which may affect your ability to have children. Talk to your healthcare provider if you have concerns about fertility.
Your healthcare provider may interrupt, reduce, or permanently stop GOMEKLI treatment if you experience certain side effects.
GOMEKLI may also cause serious side effects, including:
Tell your healthcare provider right away if you get any of the following signs or symptoms of eye problems:
Tell your healthcare provider right away if you get any of the following signs or symptoms of heart problems:
Tell your healthcare provider if you develop any of the following signs or symptoms of skin problems:
These are not all the possible side effects of GOMEKLI. Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.
During the first couple months of treatment, I experienced a rash and diarrhea, but I was able to manage my side effects by working with my doctor, applying baby lotion to the rash, and staying hydrated to help with my diarrhea.
– Austin, GOMEKLI Patient Ambassador
Use the doctor finder tool to locate a healthcare provider who has experience with NF1 near you.
This guide can help you and your healthcare provider understand if GOMEKLI could be right for you.
Available in capsules or tablets for oral suspension that give you the option to take GOMEKLI as a liquid.
GOMEKLI (mirdametinib) is a prescription medicine used to treat adults and children 2 years of age and older with neurofibromatosis type 1 (NF1) who have plexiform neurofibromas that cause symptoms and cannot be completely removed by surgery.
It is not known if GOMEKLI is safe and effective in children under 2 years of age.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
See the “Instructions for Use” that come with your medicine for instructions on how to prepare and take GOMEKLI tablets for oral suspension.
It is not known if GOMEKLI is safe and effective in children under 2 years of age.
In clinical trials, endpoints are used to measure if a treatment is working. The primary endpoint of this trial was called confirmed overall response rate (ORR).
In the GOMEKLI (mirdametinib) trial, an overall response meant that a person treated with GOMEKLI had a plexiform shrink by 20% or more. The “confirmed” part means that another scan done later also showed that the plexiform stayed smaller.
The confirmed ORR for GOMEKLI tells us how many trial participants saw their plexiform shrink by 20% or more in 2 back-to-back scans. “41% confirmed ORR” means that 24 of the 58 adults who received GOMEKLI had a plexiform shrink by at least 20% and stay smaller on the next scan.
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