GOMEKLI (mirdametinib)

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Frequently asked questions about GOMEKLI for NF1-PN

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About GOMEKLI

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 under 2 years of age.

Watch this video for some tips on how to start a conversation with your doctor about whether GOMEKLI may be right for you.

Please see Important Safety Information and Patient Information.

GOMEKLI is not traditional chemotherapy, but rather a targeted therapy called a MEK inhibitor. It works by helping to block certain signals in the body that cause plexiform neurofibromas to grow.

Learn more aboute GOMEKLI in adults and GOMEKLI in children.

Please see Important Safety Information and Patient Information.

GOMEKLI was studied in one of the largest clinical trials for NF1-PN. The single-arm trial included 114 people: 58 adults (age 18+) and 56 children and adolescents (age 2-17), including some who had undergone at least 1 PN-related surgery.

Watch this video for an overview of the GOMEKLI clinical trial, presented at the 2025 NF Summit.

Please see Important Safety Information and Patient Information.

Clinical trial results

For adults treated with GOMEKLI, the median (middle) amount of time spent on treatment was 22 months (range: 0.4 to 46 months). For children and adolescents treated with GOMEKLI, the median (middle) amount of time spent on treatment was 22 months (range: 1.6 to 40 months).

The clinical trial was about 2 years long. After that, 84% of eligible adults (26 out of 31) and 85% of eligible children and adolescents (28 out of 33) chose to keep taking GOMEKLI in an optional long-term follow-up phase of the trial. Some of those patients are still on GOMEKLI.

In February of 2025, the FDA approved GOMEKLI for use in adults and children (2 years and older) with NF1 who have PNs that cause symptoms and cannot be completely removed by surgery.

Hear about real patients who were part of the clinical trial and are still taking GOMEKLI for their NF1-PN.

Please see Important Safety Information and Patient Information.

GOMEKLI was studied and proven effective in both adults and children/adolescents in one of the largest clinical trials for NF1-PN.

41% of adults (24 out of 58) treated with GOMEKLI saw their plexiform shrink by at least 20%. See additional information on the effectiveness and safety of GOMEKLI in adults with NF1-PN.

52% of children and adolescents (29 out of 56) treated with GOMEKLI saw their plexiform shrink by at least 20%. See additional information on the effectiveness and safety of GOMEKLI in children and adolescents with NF1-PN.

GOMEKLI can cause serious side effects, including eye problems, heart problems, and skin problems.

Most side effects were mild to moderate. The most common side effects with GOMEKLI were diarrhea; muscle, joint, and bone pain; stomach (abdominal) pain; vomiting; headache; skin redness, swelling, or pain around the fingernails or toenails; tiredness; and nausea.

These are not all the possible side effects of GOMEKLI.

Please see Important Safety Information and Patient Information.

In the clinical trial, patients who benefited from GOMEKLI saw their plexiforms begin to shrink by at least 20% within 4 to 19 months of starting treatment. In fact, of those who benefited, 46% of adults (11 out of 24) and 45% of children and adolescents (13 out of 29) started to see a response after about 4 months.

See additional results in adults and additional results in children/adolescents.

Please see Important Safety Information and Patient Information.

In the clinical trial, the most common side effects associated with GOMEKLI in adults were diarrhea; nausea; muscle, joint, and bone pain; vomiting; and tiredness. Learn more about possible side effects in adults.

The most common side effects associated with children and adolescents were diarrhea; muscle, joint, and bone pain; stomach (abdominal) pain; vomiting; headache; nausea; and skin redness, swelling, or pain around the fingernails or toes. Learn more about possible side effects in children/adolescents.

Watch this video on how to manage possible side effects with the help of your care team.

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 the FDA at 1-800-FDA-1088.

GOMEKLI may also cause serious side effects. Please see Important Safety Information and Patient Information.

Taking GOMEKLI

GOMEKLI is taken by mouth twice a day (about 12 hours apart), with or without food. Each GOMEKLI dosing cycle is 28 days (4 weeks). GOMEKLI is taken for the first 21 days, followed by 7 days when GOMEKLI is not taken. The next cycle then begins with 21 days of treatment.

Watch this video about how to stay on top of treatment with your own easy-to-use GOMEKLI dosing calendar.

Please see Important Safety Information and Patient Information.

Yes. GOMEKLI comes in 2 forms: capsules that come in 2 sizes and grape-flavored tablets for oral suspension. Oral suspension means the tablets mix or disperse into water, so the medicine can be taken as a liquid. This may make taking GOMEKLI easier for people who have difficulty swallowing. Get a closer look at the dosing options available for GOMEKLI.

View a step-by-step guide on how to prepare GOMEKLI as a liquid.

Please see Important Safety Information and Patient Information.

If you miss a dose of GOMEKLI, take your next dose at your regular time. You should not take 2 doses of GOMEKLI to make up for the missed dose. Talk to your healthcare provider if you have questions about dosing.

Watch this video for tips on how to stay on top of GOMEKLI treatment over time.

Additionally, the GOMEKLI Digital Companion is a free resource that you can use to set treatment reminders to help you remember your dose.

Please see Important Safety Information and Patient Information.

As with any medication, results vary from one person to another. Your healthcare provider may keep you on GOMEKLI treatment for as long as it continues to work for you, and may interrupt, reduce, or permanently stop GOMEKLI treatment if you experience certain side effects. In the clinical trial, adults were on GOMEKLI between 0.4 and 46 months and children and adolescents were on GOMEKLI between 1.6 and 40 months. Your healthcare provider will decide how many treatment cycles are right for you.

Hear about real patients who were part of the clinical trial and are still taking GOMEKLI for their NF1-PN.

Please see Important Safety Information and Patient Information.

Patient support

SpringWorks CareConnections® is a free, personalized patient support program. It can help you—or the person you care for—navigate insurance coverage, get answers to questions about treatment, and get started and stay on track with GOMEKLI.

Watch this video to learn more about the SpringWorks CareConnections patient support program.

Once your doctor prescribes GOMEKLI, enrolling in SpringWorks CareConnections takes a few short steps.

With the SpringWorks CareConnections® Commercial Copay Program, commercially insured patients may pay as little as a $0 copay per 21-day supply of GOMEKLI.* The program may also help with eligible out-of-pocket costs for certain treatment-related tests, examinations, and/or specialty visits during treatment with GOMEKLI.  

Learn more about insurance navigation and financial help available through SpringWorks CareConnections.

*Terms and conditions apply. Copay program for GOMEKLI and reimbursement for eligible treatment-related costs are subject to annual benefit maximums. To receive the reimbursement of eligible treatment-related expenses, an Explanation of Benefits (EOB) form must be submitted, along with copies of receipts for any payments made in full, or your healthcare provider may submit an invoice for services rendered. Full terms and conditions are provided during the enrollment process and are available upon request by contacting SpringWorks CareConnections at 844-CARES-55 (844-227-3755).

Helpful resources

GOMEKLI offers a variety of resources to help you or the person you care for get started and stay on track with GOMEKLI. Explore downloadable tools and helpful videos designed to inform and guide you on your journey. If you have been prescribed GOMEKLI, you can also connect with someone else who is on treatment and hear about their experience through the GOMEKLI Patient Mentor Program.†

†To be eligible to participate in the program, you must be at least 18 years of age, reside in the United States, and have, or care for someone who has, a valid prescription for GOMEKLI from a healthcare provider licensed in the United States for an FDA-approved indication.

If you're looking for a healthcare provider with NF1 experience, you can use this tool to locate an NF1 treater near you. 

The Children's Tumor Foundation, NF Network, and NF Collective websites also feature tools that can help you locate healthcare providers throughout the US who are familiar with NF1.‡

‡SpringWorks Therapeutics is providing these independent resources to help patients find an NF1 treater by region, but SpringWorks Therapeutics had no role in their development. Their inclusion on this website does not represent an endorsement or a recommendation from SpringWorks Therapeutics for any center or physician.

Additional questions?

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NF1-PN=neurofibromatosis type 1 with plexiform neurofibromas.

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 (PN) 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 breastmilk.
    • 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 (PN) 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.