Neurofibromatosis type 1 with plexiform neurofibromas (NF1-PN) is a life-changing condition
Actor portrayal.
What is NF1-PN?
- Neurofibromatosis type 1 (NF1) is a genetic condition that affects about 1 in 2500 babies born worldwide
- One common type of tumor associated with NF1 is known as a plexiform neurofibroma (PN), which can simply be called a plexiform
- NF1 is a chronic disease that is not curable, but PNs can be manageable
Up to
50%
of people living with NF1 develop plexiforms
Plexiforms can grow along nerves anywhere in the body except in the brain and spinal cord
From one person to another, plexiforms vary in:
Size
Number
Location
Find the care you need
Use the doctor finder tool to locate a healthcare provider near you who has experience with NF1.
People who don’t have NF1-PN may not understand how it can affect the body
The most common symptoms of NF1-PN include:
Pain
Difficulty moving around
Changes in appearance
In one study, 93% of adolescents with NF1-PN and 100% of caregivers reported pain as interfering with daily functioning, even when pain medications were used.*
- Because plexiforms can press against internal organs or grow into surrounding tissues, people with NF1-PN may also experience a variety of symptoms, including problems with their hearing, vision, or breathing
- In some cases it is possible for plexiforms to become cancerous
*Based on a questionnaire completed by the caregivers of 59 people with NF1-PN aged 6 to 18, and 41
people with NF1-PN aged 10 to 18.
Nonsurgical treatment options for NF1-PN are needed
- Surgery may be an option in some cases, but even when it is possible, up to 85% of plexiforms cannot be completely removed by surgery
- There are other factors to consider with surgery, including recovery time
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.
Learn about the first NF1-PN treatment approved for both adults and children
NF organizations‡
If you’re living with or caring for someone with NF1-PN, there are a number of advocacy groups where you can get information or connect with others in the NF community.
The NF Collective is a group of organizations dedicated to improving the lives of NF patients and their families by providing accurate and reliable information about NF resources and caregivers. The Children’s Tumor Foundation and Littlest Tumor Foundation are part of the NF Collective.
Independent Patient Assistance Foundations
Financial assistance may be available through independent foundations that are not controlled by or otherwise affiliated with SpringWorks Therapeutics. These foundations may be able to assist with copays, premiums, patient education, social services, etc.
SpringWorks Therapeutics does not influence or control the operations of independent patient assistance foundations and cannot guarantee assistance will be provided. Details and eligibility requirements can be found directly on the individual foundation websites.
Explore additional foundations that may be able to help with financial assistance
‡SpringWorks Therapeutics is providing these websites and resources to help patients find more information about NF1-PN, but their inclusion does not represent an endorsement or a recommendation from SpringWorks Therapeutics for any group or organization. Any organization listed is independent of SpringWorks Therapeutics.
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.