What treatment options are available for people with NF1-PN?
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Understanding your treatment options
People living with NF1‑PN may have several treatment options to consider. These can include targeted therapies—such as GOMEKLI—as well as surgery in certain cases. Your treatment plan will depend on factors like the size and location of your plexiform and the symptoms you’re experiencing. Working closely with your healthcare provider can help you choose the option that’s right for you.
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What is targeted therapy for NF1-PN?
There are treatments approved for people who have plexiforms that are causing symptoms and cannot be completely removed by surgery. They’re known as targeted treatments because they help to block certain signals in the body that cause plexiforms to grow.
GOMEKLI is a targeted therapy called a MEK inhibitor that has been shown to shrink the size of plexiforms in both adults and children with NF1-PN.
My one-year appointment after starting GOMEKLI marked a turning point for me. For years, I had seen my NF1-PN as a burden. But hearing that my tumor was shrinking, I felt like I had a completely new outlook.
– Austin, GOMEKLI Patient Ambassador
Individual results may vary.
Learn more about the GOMEKLI clinical trial results.
When is surgery an option for treating NF1-PN?
For many years, surgery has been the standard treatment for NF1-PN; however, it does come with challenges.
- Due to the size and location of some PNs, complete removal may not always be possible
- When complete removal is not possible, partial removal (called “debulking”) may be performed, but there are other factors to consider, including recovery time
- Additionally, there is a chance that a plexiform will regrow after surgery
Up to 85% of plexiforms cannot be completely removed by surgery
Nearly 1 in 4 plexiforms regrow after surgery*
*In a study of medical records of 52 children and adults with NF1 who had surgery to remove plexiforms.
At four and a half years old, I was undergoing my first major surgery to have part of a tumor removed. Then, just 10 months later, I had to undergo a second surgery because my tumor had started to grow again in the same area, this time to the size of a golf ball.
– Austin, GOMEKLI Patient Ambassador
Why is routine monitoring important in NF1-PN?
With a condition like NF1-PN, regular appointments with your healthcare provider are so important, even if you’re not experiencing symptoms. Whether you’re on treatment or not, these check-ins give your healthcare provider a chance to assess for changes.
Depending on the size and location of your plexiform, and whether or not it’s causing symptoms, you and your doctor may decide the best course of action is monitoring the plexiform over time. This is sometimes referred to as “watch and wait.”
If you’re on a watch-and-wait plan, it’s important that you stay actively engaged in your care. Reach out to your healthcare provider about any new or worsening symptoms you experience, including:
Pain on or around your plexiform
Difficulty moving around
Changes in appearance
No one knows your body better than you do, so if something changes, it’s important that you speak up.
Find the care you need
Use the doctor finder tool to locate a healthcare provider near you who has experience with NF1.
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.
- Disperse the tablets for oral suspension in drinking water to make a liquid (suspension) before you take or give GOMEKLI.
OR
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.