Gomekli
Gomekli (mirdametinib) is an FDA‑approved medication for neurofibromatosis type 1 (NF1). It's used for people ages 2 years and up with NF1 who have tumors called plexiform neurofibromas (PN) that can't be fully removed with surgery. Gomekli (mirdametinib) works as a MEK inhibitor to shrink the tumors. It comes as capsules as well as tablets that can be swallowed or mixed in water. You take the medication by mouth twice a day for 21 days, then have 7 days off in a repeating 28‑day cycle. Common side effects include rash, diarrhea, and nausea.
What is Gomekli (mirdametinib)?
What is Gomekli (mirdametinib) used for?
- Neurofibromatosis type 1 (NF1) with symptomatic plexiform neurofibromas (PN) that can’t be fully removed by surgery
How Gomekli (mirdametinib) works
Gomekli (mirdametinib) is a MEK inhibitor. It blocks proteins in the body called MEK. These proteins are part of a signaling pathway that's too active in people with NF1. This overactive signaling causes too much cell growth and leads to tumors called plexiform neurofibromas (PN).
By blocking MEK proteins, Gomekli (mirdametinib) disrupts this signaling pathway. This helps shrink the tumors or keep them from growing.
Drug facts
| Common Brands | Gomekli |
|---|---|
| Drug Class | MEK inhibitor |
| Controlled Substance Classification | Not a controlled medication |
| Generic Status | No lower-cost generic available |
| Availability | Prescription only |
Side effects of Gomekli (mirdametinib)
The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.
Common Side Effects
- Rash (82%)
- Diarrhea (57%)
- Higher creatine phosphokinase levels (57%)
- Joint, muscle, or bone pain (41%)
- Nausea (40%)
- Vomiting (39%)
- Higher triglyceride levels (37%)
- Stomach pain (32%)
- Lower hemoglobin levels (25%)
Less Common Side Effects
- Headache
- Tiredness
- Cough
- Fever
- Mouth sores
- Numbness or tingling in the arm or leg
- Constipation
- Blurry vision
- Hair loss
- Hair color changes
- COVID-19 infection
- Upper respiratory tract infection
- Nail infection
- Lower heart function
- Lower calcium levels
- Higher creatinine levels
- Higher liver enzyme levels
- Higher cholesterol
Gomekli (mirdametinib) serious side effects
Contact your healthcare provider immediately if you experience any of the following.
- Eye problems: blurry vision, new floaters, flashes of light, dark spots, eye pain, vision loss
- Lower heart function or weaker heart: shortness of breath, cough, wheezing, tiredness, chest discomfort, leg or ankle swelling, sudden weight gain, faster heart rate
- Severe skin reactions: widespread or worsening rash, blistering or peeling skin, painful sores
The following Gomekli (mirdametinib) side effects have also been reported
Side effects that you should report to your care team as soon as possible:
- Allergic reactions—skin rash, itching, hives, swelling of the face, lips, tongue, or throat
- Blurry vision, vision loss, seeing wavy or bent objects or blind spots with dark, light, or flashing spots
- Heart failure—shortness of breath, swelling of the ankles, feet, or hands, sudden weight gain, unusual weakness or fatigue
- Redness, swelling, and blistering of the skin over hands and feet
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
- Diarrhea
- Fatigue
- Hair loss
- Headache
- Muscle pain
- Nausea
- Vomiting
Pros and cons of Gomekli (mirdametinib)
Pros
- Taken by mouth
- Also available as tablets for oral suspension that can be dissolved in water if swallowing pills is hard
- Can help shrink PN in many people with NF1
Cons
- Taken twice a day on a 3-weeks-on, 1-week-off schedule that you must follow closely
- Often causes skin rash
- Need regular eye exams and heart checks for safety
Pharmacist tips for Gomekli (mirdametinib)
- You can take Gomekli (mirdametinib) with or without food.
- Stick to your dosing schedule for Gomekli (mirdametinib). You take it twice a day, about 12 hours apart, for 3 weeks, then you take a week break in a repeating 4-week cycle. Set reminders and use a calendar to help you remember. Ask your care team to review your treatment plan with you.
- If you miss a dose of Gomekli (mirdametinib), skip it. Wait to take your usual dose the next time you're supposed to. Don't take an extra dose to make up for the missed one because this can raise your risk of side effects.
- If you vomit after taking Gomekli (mirdametinib), don't take an extra dose. Wait for the next time you usually take the medication to take your usual dose.
- Look out for any skin changes while you're taking Gomekli (mirdametinib). Rash is very common. Use gentle moisturizer, avoid harsh skin products, and tell your care team early at the first sign of a rash. They can help manage skin reactions and help keep them from getting severe.
- Nausea, diarrhea, and mouth sores can happen with Gomekli (mirdametinib). Drink plenty of fluids and eat small meals to help. If needed, ask your care about anti-nausea medications, anti‑diarrheals, or mouthwashes that might help. Managing these side effects is important so you can stay on track with treatment.
- If you have blurry vision, see floaters, or have other vision changes while taking Gomekli (mirdametinib), tell your care team. This medication can affect your vision. Catching problems early can help prevent more serious issues.
- Be sure to go to all your lab appointments, eye exams, and echocardiograms while you're taking Gomekli (mirdametinib). This helps your NF1 specialist make sure the medication stays safe for you.
- If you can become pregnant, talk to your prescriber about family planning. Gomekli (mirdametinib) can harm an unborn baby. It can also make it harder to become pregnant. Use reliable birth control while taking this medication and for 6 weeks after stopping it.
- If your partner can become pregnant, use birth control like condoms during sex while taking Gomekli (mirdametinib). Keep using birth control for 3 months after your last dose. This medication can cause harm to an unborn baby.
Tips for the Gomekli (mirdametinib) capsules:
- Swallow the capsule whole with drinking water. Don’t open, break, or chew them.
- If you need to take more than 1 capsule for each dose, swallow 1 capsule at a time. This helps prevent choking.
Tips for the Gomekli (mirdametinib) tablets for oral suspension:
- You can swallow the tablet whole with drinking water. If you need to take more than 1 for each dose, swallow 1 tablet at a time to avoid choking.
- If you have trouble swallowing pills, you can mix the tablet with water to make a liquid. Add the number of tablets for your dose into a cup with about 1 to 2 teaspoons (5 mL to 10 mL) of drinking water. Gently swirl until the tablet(s) dissolve and the liquid looks white and cloudy. This should take about 2 to 4 minutes. Drink right away or use an oral syringe to take it. Then, add a little more water, swirl, and drink again to get the full dose. Take the liquid within 30 minutes of mixing.
Risks and warnings for Gomekli (mirdametinib)
Gomekli (mirdametinib) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Eye problems
Gomekli (mirdametinib) can cause eye problems. This can include blurry vision and problems with the retina. Most cases are mild, but they can also be serious.
Before starting Gomekli (mirdametinib), you’ll need a full eye exam. You'll also need periodic checks during treatment and extra eye exams if your vision changes.
Tell your NF1 care team right away about any new or worse vision changes while taking Gomekli (mirdametinib). These include blurry vision, new floaters, flashes, dark spots, or loss of vision. Depending on your eye symptoms, you might need to pause, take a lower dose, or stop the medication.
Lower heart function
- Risk factors: History of heart disease | Lower ejection fraction
Gomekli (mirdametinib) can lower how well your heart pumps blood. This can show up on heart scans called echocardiograms (echoes). In studies, this sometimes happened a few months after starting the medication. Most of time time, it gets better over time.
Before you start Gomekli (mirdametinib), your care team will check your heart with an echo. They'll also check it every 3 months during your first year of treatment and then when needed. If there's a problem, your prescriber might pause treatment, lower your dose, or stop Gomekli (mirdametinib).
Some people might not notice symptoms of having lower heart function. But watch for shortness of breath, cough, swelling in your legs, sudden weight gain, or unusual tiredness while taking Gomekli (mirdametinib). If you notice these symptoms, tell your care team so they can check your heart.
Skin reactions, including rash
Skin reactions are very common with Gomekli (mirdametinib). Rash happens often, and they can sometimes be acne-like or eczema-like.
If you notice a new or worse rash while taking Gomekli (mirdametinib), start gentle skin care early and tell your care team right away. They can help treat the rash. Depending on how severe it is, your prescriber might pause Gomekli (mirdametinib), lower your dose, or stop it.
Get medical help right away if you notice blistering, peeling, or painful sores. These can be signs of a severe reaction.
Harm to an unborn baby
- Risk factors: Currently pregnant | People who can become pregnant
Gomekli (mirdametinib) can harm an unborn baby based on how it works and studies in animals and people. If you can become pregnant, you’ll need a pregnancy test before starting to make sure it's safe.
If you can become pregnant, use effective birth control during treatment and for 6 weeks after your last dose. If your partner can become pregnant, use birth control like condoms during sex while you're taking Gomekli (mirdametinib) and for 3 months after stopping it. If you or your partner might be pregnant during treatment, call your care team right away.
Gomekli (mirdametinib) dosage
Typical dosage for Gomekli (mirdametinib)
In general, the dose is taken every day for the first 21 days. Then, you take a break for the next 7 days to finish each 28-day treatment cycle. Repeat this dosing schedule until you stop treatment.
Your NF1 specialist will help determine your dose. It’s based on your body size or body surface area (BSA). This is measured in m² units and it takes into account your height and weight.
- BSA of 0.4 m² to 0.69 m²: The typical dose is 1 mg by mouth twice a day (about every 12 hours). Take it for the first 21 days of each cycle.
- BSA of 0.7 m² to 1.04 m²: The typical dose is 2 mg by mouth twice a day (about every 12 hours). Take it for the first 21 days of each cycle.
- BSA of 1.05 m² to 1.49 m²: The typical dose is 3 mg by mouth twice a day (about every 12 hours). Take it for the first 21 days of each cycle.
- BSA of 1.5 m² or greater: The typical dose is 4 mg by mouth twice a day (about every 12 hours). Take it for the first 21 days of each cycle.
Your prescriber might lower your dose based on any side effects you have.
