STAMFORD, Conn., March 02, 2026 (GLOBE NEWSWIRE) -- Did you know neurofibromatosis type 1 with plexiform neurofibromas (NF1-PN) is a rare genetic disorder that can cause tumors to grow along nerves, leading to pain, mobility challenges, and lifelong complications?1 This rare condition affects approximately 30,000 to 50,000 people in the United States, including Austin, who has lived with NF1-PN since early childhood.1-5
A Media Snippet accompanying this announcement is available by clicking on this link.
Living with NF1-PN has affected nearly every aspect of Austin’s life, from repeated surgeries at a young age, to pain and other symptoms which forced him to give up his career in law enforcement. As he grew older, Austin learned to advocate for himself, working closely with his doctors to manage symptoms and explore treatment options. After consulting with his care team, Austin decided to participate in a clinical trial for what is now FDA-approved as GOMEKLI® (mirdametinib) for treating symptomatic, inoperable PNs in adults and children with NF1. Please see important safety information and Patient Information for GOMEKLI.
Austin shares his experience to help others feel less alone and encourage those newly diagnosed to speak up, seek specialized care, and connect with the NF1-PN community for more information and resources.
Learn more: https://www.gomekli.com/
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:
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- 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:
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- 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.
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- 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:
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- Swallow each tablet for oral suspension whole with drinking water. If more than 1 tablet is required, swallow 1 tablet at a time.
OR
- 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.
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:
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- 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:
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- 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:
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- 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:
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- 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.
Please click here for full Prescribing Information including Patient Information and Instructions for Use.
References:
- Ejerskov, C., Farholt, S., Nielsen, F.S.K. et al. Clinical Characteristics and Management of Children and Adults with Neurofibromatosis Type 1 and Plexiform Neurofibromas in Denmark: A Nationwide Study. Oncol Ther 11, 97–110 (2023). https://doi.org/10.1007/s40487-022-00213-4.
- Prada CE, Rangwala FA, Martin LJ, et al. Pediatric plexiform neurofibromas: impact on morbidity and mortality in neurofibromatosis type 1. J Pediatr. 2012;160(3):461-467.
- Miller DT, Freedenberg D, Schorry E, Ullrich NJ, Viskochil D, Korf BR; Council on Genetics; American College of Medical Genetics and Genomics. Health supervision for children with neurofibromatosis type 1. Pediatrics. 2019;143(5):e20190660.
- Kallionpää RA, Uusitalo E, Leppävirta J, Pöyhönen M, Peltonen S, Peltonen J. Prevalence of neurofibromatosis type 1 in the Finnish population. Genet Med. 2018;20(9):1082-1086.
- U.S. population estimated at 332,403,650 on Jan. 1, 2022. U.S. Department of Commerce. January 6, 2022. Accessed April 16, 2024. https://www.commerce.gov/news/blog/2022/01/us-population-estimated-332403650-jan-1-2022.
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