GOMEKLI (mirdametinib)

Menu

GOMEKLI is proven to help kids
with NF1-PN

Actor portrayal.

What is GOMEKLI (mirdametinib)?

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. 

Image of an actor portraying a child patient with NF1-PN

GOMEKLI has been proven to shrink plexiforms in children and adolescents (age 2-17) with NF1-PN

The clinical trial enrolled 58 adults (age 18+) and 56 children/adolescents (age 2-17), including some who already had undergone at least 1 PN-related surgery. At the start of the trial, pain and changes in appearance were the most common PN-related symptoms reported by adults and children.

52% of children/adolescents

(29 out of 56) treated with GOMEKLI

SAW THEIR PLEXIFORM SHRINK by at least 20%*

15 of these 
children/adolescents

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.

""

Plexiform is short for plexiform neurofibroma, which can also be called a PN.

Why 20%?

The 20% change in plexiform volume is the standard benchmark used in NF1-PN clinical trials.
""
GOMEKLI is helping to treat my son, Jacoby. Since starting the treatment as part of the clinical trial, his MRI scans have shown a 50% decrease in the size of his plexiform.

– Julie, GOMEKLI Caregiver Ambassador

Individual results may vary.

In children/adolescents who benefited from treatment, plexiforms started to shrink within months of taking GOMEKLI

Image showing when pediatric patients treated with GOMEKLI™ (mirdametinib) saw their plexiform get smaller
Image showing when pediatric patients treated with GOMEKLI™ (mirdametinib) saw their plexiform get smaller

In children/adolescents 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 7.9 months.

The GOMEKLI clinical trial was about 2 years long. After that,
85% of children/adolescents chose to keep taking GOMEKLI in an optional long-term follow-up phase of the study.

Possible side effects with GOMEKLI (mirdametinib)

In children and adolescents, the most common ones were:
  • Diarrhea
  • Muscle, joint, and bone pain
  • Stomach (abdominal) pain
  • Vomiting
  • Headache
  • Nausea
  • Skin redness, swelling, or pain around the fingernails or toenails
The most common severe abnormal blood test results in children and adolescents involved decreased white blood cell counts and an increased enzyme called creatine phosphokinase (CPK).

Most children/adolescents taking GOMEKLI did not have to stop treatment due to a side effect

""

had to stop treatment due to a side effect

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:

""
Eye problems

Tell your healthcare provider right away if you get any of the following signs or symptoms of eye problems:

  • Blurred vision, loss of vision, or other changes to your vision
""
Heart problems

Tell your healthcare provider right away if you get any of the following signs or symptoms of heart problems:

  • Coughing or wheezing, shortness of breath, swelling of your ankles and feet, tiredness, increased heart rate
""
Skin problems

Tell your healthcare provider if you develop any of the following signs or symptoms of skin problems:

  • Flat skin rash, raised bumps on the skin, skin bumps that look like acne, skin redness, itchy rash, peeling skin
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 Jacoby’s treatment, he experienced headaches and nausea. To get ahead of this, we gave him an over-the-counter pain reliever 1 to 2 days before his treatment-free week, which helped him manage the side effects he was experiencing.

– Julie, GOMEKLI Caregiver Ambassador

Individual results may vary.
Image of a healthcare provider. Locate a doctor who has experience with NF1 near you.

Find the care you need

Use the doctor finder tool to locate a healthcare provider who has experience with NF1 near you.

Start searching
""

Is GOMEKLI right for me?

This guide can help you and your healthcare provider understand if 
GOMEKLI could be right for you.

Download guide
""

Dosing that fits your needs

Available in capsules or tablets for oral suspension that give you the option to take GOMEKLI as a liquid.

Learn more

What is GOMEKLI?

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.

Important Safety Information

Before taking GOMEKLI, tell your healthcare provider about all of your medical conditions, including if you:

  • Have eye problems
  • Have heart problems
  • Are pregnant or plan to become pregnant. GOMEKLI can harm your unborn baby

    Females who are able to become pregnant:

    • Your healthcare provider should check to see if you are pregnant before you begin treatment with GOMEKLI.
    • Use effective birth control (contraception) during treatment with GOMEKLI and for 6 weeks after your last dose.
    • Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with GOMEKLI.

    Males with female partners who are able to become pregnant:

    • Use effective birth control (contraception) during treatment with GOMEKLI and for 3 months after your last dose.
    • Tell your healthcare provider right away if your female partner becomes pregnant or thinks she may be pregnant during treatment with GOMEKLI.
  • Are breastfeeding or plan to breastfeed. It is not known if GOMEKLI passes into your breast milk.
    • Do not breastfeed during treatment with GOMEKLI and for 1 week after your last dose.
    • Talk to your healthcare provider about the best way to feed your baby during this time.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

How should I take GOMEKLI?

  • Take GOMEKLI exactly as your healthcare provider tells you to take it. Your healthcare provider may change your dose, temporarily stop, or permanently stop treatment with GOMEKLI if you develop certain side effects.
  • Take GOMEKLI twice a day, about 12 hours apart, for 21 days, followed by 7 days off treatment, to complete a 28-day treatment cycle. Your healthcare provider will decide how many treatment cycles are right for you.
  • Take GOMEKLI with or without food.
  • GOMEKLI comes in two different dosage forms, GOMEKLI capsules and GOMEKLI tablets for oral suspension. Your healthcare provider will decide the dosage form and dose of GOMEKLI that is right for you.
  • If you take GOMEKLI capsules: Swallow each capsule whole with drinking water. If more than 1 capsule is required, swallow 1 capsule at a time. Do not open, break or chew the capsules.
  • If you take GOMEKLI tablets for oral suspension, either:
    • Swallow each tablet for oral suspension whole with drinking water. If more than 1 tablet is required, swallow 1 tablet at a time.

      • OR

    • Disperse the tablets for oral suspension in drinking water to make a liquid (suspension) before you take or give GOMEKLI.

    See the “Instructions for Use” that come with your medicine for instructions on how to prepare and take GOMEKLI tablets for oral suspension.

  • If you miss a dose of GOMEKLI, skip the missed dose and take your next dose at your regularly scheduled time.
  • If you vomit at any time after taking GOMEKLI, do not take an additional dose. Take your next dose at your regularly scheduled time.

What are the possible side effects of GOMEKLI?

GOMEKLI may cause serious side effects, including:
  • Eye problems. GOMEKLI may cause eye problems that can lead to blindness. Your healthcare provider will check your vision before and during treatment with GOMEKLI. Tell your healthcare provider right away if you get any of the following signs or symptoms of eye problems:
    • Blurred vision
    • Loss of vision
    • Other changes to your vision
  • Heart problems. GOMEKLI may lower the amount of blood pumped by your heart, which is common in children during treatment with GOMEKLI and can also be severe. Your healthcare provider will do tests before you start GOMEKLI treatment, every 3 months during your first year of treatment, and then as needed to make sure your heart is working properly. Tell your healthcare provider right away if you get any of the following signs or symptoms of heart problems:
    • Coughing or wheezing
    • Shortness of breath
    • Swelling of your ankles and feet
    • Tiredness
    • Increased heart rate
  • Skin problems. Skin rashes are common with GOMEKLI in both adults and children and can also be severe. GOMEKLI can also cause hair loss (alopecia). Tell your healthcare provider if you develop any of the following signs or symptoms of skin problems:
    • Flat skin rash
    • Raised bumps on the skin
    • Skin bumps that look like acne
    • Skin redness
    • Itchy rash
    • Peeling skin

The most common side effects of GOMEKLI in adults include:

  • Diarrhea
  • Nausea
  • Muscle, joint, and bone pain
  • Vomiting
  • Tiredness
The most common severe abnormal blood tests in adults include an increased enzyme called creatine phosphokinase (CPK).

The most common side effects of GOMEKLI in children include:

  • Diarrhea
  • Muscle, joint, and bone pain
  • Stomach (abdominal) pain
  • Vomiting
  • Headache
  • Skin redness, swelling, or pain around the fingernails or toenails
  • Nausea
The most common severe abnormal blood tests in children include decreased white blood cell (neutrophil) counts and increased 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.
These are not all of the possible side effects of GOMEKLI. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

What is GOMEKLI?

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.

Please click here for full Prescribing Information, including Patient Information and Instructions for Use.
Please click here for full Prescribing Information, including Patient Information and Instructions for Use.