GOMEKLI (mirdametinib) has been proven effective in children with NF1-PN
Actor portrayal.
What is 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 younger than 2 years of age.
GOMEKLI is a MEK inhibitor, which is targeted therapy—not traditional chemotherapy. It works by helping to block certain signals in the body that cause plexiform neurofibromas to grow.
Watch this video for tips on how to talk to your doctor about GOMEKLI.
GOMEKLI has been proven to shrink plexiforms in children and adolescents (age 2-17) with NF1-PN
GOMEKLI was studied in one of the largest (N=114) clinical trials for neurofibromatosis type 1 with plexiform neurofibromas (NF1-PN).
The single-arm trial enrolled 58 adults (age 18+) and 56 children/adolescents (age 2-17), including some who already had undergone at least 1 PN-related surgery. At the start of the trial, pain and changes in appearance were the most common PN-related symptoms reported by adults and children.
52% of children/adolescents
(29 out of 56) treated with GOMEKLI
SAW THEIR PLEXIFORM SHRINK by at least 20%*
15 of these children/adolescents
SAW THEIR PLEXIFORM SHRINK
by more than 50%†
Plexiform before starting GOMEKLI
20%
SMALLER
50%
SMALLER
*Reduction in plexiform size had to be confirmed on a later magnetic resonance imaging (MRI) scan.
†The 50% reduction in plexiform size was an exploratory analysis, meaning it was not specifically defined in advance of the trial.
Plexiform is short for plexiform neurofibroma, which can also be called a PN.
Meet Julie and Jacoby
Meet Julie and Jacoby
Hear Julie and her son Jacoby talk about his experience on GOMEKLI treatment.
Image onscreen:
We see Jacoby sitting casually on a carpeted floor, leaning back against an armchair as he strums an electric guitar that is plugged into an amplifier. He is in a living area of a home with two armchairs, a table with a frame and houseplant, and artwork on the walls. Closeup shot of Jacoby’s left hand playing chords on the neck of the guitar.
Julie:
Jacoby has always been this bright, happy kid full of energy, full of curiosity. He loves music, sports, and just being a kid.
Image onscreen:
Julie is seated in an interview chair, talking to an interviewer off-camera. The background shows a living space with an armchair, a window seat with pillows, a piano, and a side table with stacked books. A name super graphic appears onscreen.
Text onscreen:
Julie
Mother and caregiver to Jacoby living with NF1-PN and taking GOMEKLI (mirdametinib) Julie is being compensated by SpringWorks Therapeutics, Inc.
Julie:
But behind all of that, there’s this part of his story that has shaped so much of our lives – his journey with neurofibromatosis type 1 with plexiform neurofibromas or NF1-PN.
Image onscreen:
Jacoby makes an adjustment on his amplifier by turning a couple knobs. He then continues to strum his guitar. Closeup shots of his hands and face as he plays.
Julie:
His dad and I first noticed something when Jacoby was just a few months old. He had this cute coffee colored mark on his belly. At first, we didn’t think much of it and thought it might just be a birthmark, but as we started paying more attention, we noticed more of them.
Image onscreen:
Julie is seated in an interview chair, talking to at an interviewer off-camera.
Julie:
We didn’t know what they meant. I did what most new parents would do – I searched the internet. I read that Jacoby’s marks might be something called café-au-lait spots.
Image onscreen:
We see a shot of a blue-colored lava lamp, with bubbles slowly morphing inside the lamp. The video then switches back to Jacoby as he makes some adjustments on the tuning knobs on the head of his guitar. He continues to strum the guitar, then gets up, sets his guitar down on a stand, and unplugs a cable from the amplifier.
Julie:
And that’s the first time I came across neurofibromatosis type 1, or NF1. I remember reading NF1 is a genetic disorder that can cause non-cancerous tumors to grow along nerves throughout the body.
Image onscreen:
Julie is seated in an interview chair, talking to at an interviewer off-camera.
Julie:
My heart sank. No one in our family had ever faced something like this. It wasn’t something that had ever been on our radar. But my gut told me something was wrong, and I pushed for answers.
Image onscreen:
Julie is seated on a large corner couch. The wall behind has wood wainscoting and there is a round coffee table with a tray and candle in front of Julie. She holds a photo album in her lap. Jacoby walks into frame and joins her on the couch. Together, they look through the photo album and point and reminisce over family photos.
Julie:
I brought it up to our pediatrician, but she wasn’t familiar with NF1 and initially wasn’t too concerned. She did some research, made some calls, and got us connected with the NF1 clinic at the local university. The clinic took a sample of Jacoby’s blood to run tests. When the results came back, we got the official diagnosis of NF1.
Image onscreen:
Julie is seated in an interview chair, talking to at an interviewer off-camera.
Julie:
We were scared. We didn’t know what to do. The NF1 team told us all we could do was wait and see how Jacoby’s symptoms progressed.
Image onscreen:
Julie and Jacoby continue to look through the photo album.
Julie:
For a while life felt normal. Jacoby was happy, active, hitting all of his milestones. But then, around five-years-old, he started waking up in the middle of the night with intense ankle pain. Doctors told us NF1 patients often experience pain like this, and they didn’t think it was a tumor. And then one day I pressed on his abdomen, and he jolted in pain.
Image onscreen:
Julie is seated in an interview chair, talking to at an interviewer off-camera.
Julie:
I knew something was wrong. I didn’t want my son to keep experiencing this pain and discomfort. That's when I demanded imaging.
Image onscreen:
Julie and Jacoby continue to look through the photo album.
Julie:
Jacoby’s scan showed a large plexiform neurofibroma, or PN, that could not completely be removed by surgery. It had been growing all along pressing on nerves in his abdomen and groin.
Image onscreen:
Julie is seated in an interview chair, talking to at an interviewer off-camera.
Julie:
That’s when we realized that we weren’t just dealing with NF1. We were dealing with NF1-PN. Jacoby’s tumor could keep growing, causing pain, affecting his mobility. It was terrifying.
Image onscreen:
Jacoby is seated on a bench in an ice-hockey arena. He is talking to his father, who is seated on the bench next to him. We see hockey equipment on the bench next to Jacoby. Jacoby then puts on his ice skates. Closeup shot of Jacoby tying his skate laces. A disclaimer graphic appears along the bottom of the screen.
Julie:
But our NF doctor was incredible. The moment he showed us the scan he said, “There’s a clinical trial and I think Jacoby would be perfect for it.” That’s how we first heard about mirdametinib, what is now GOMEKLI.
Text onscreen:
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.
Image onscreen:
Julie is seated in an interview chair, talking to at an interviewer off-camera.
Julie:
We talked with Jacoby’s doctor about what to expect during the trial.
Image onscreen:
Closeup shot of Jacoby grabbing his gloves off the bench. We then see Jacoby putting on his helmet and mask. He then grabs his hockey stick.
Julie:
His doctor said the treatment goal was to reduce the size of his plexiform neurofibroma by at least 20%. We trusted our doctor and wanted to do whatever we could to help Jacoby.
Image onscreen:
Julie is seated in an interview chair, talking to at an interviewer off-camera.
Julie:
We decided we wanted to be in this trial.
Image onscreen:
Jacoby and his father stand up from the bench and walk along the edge of the rink. A disclaimer graphic appears along the bottom of the screen.
Text onscreen:
Please see Important Safety Information throughout and Full Prescribing Information including Patient Information.
Julie:
We hoped with the reduction of the tumor that Jacoby might get some relief from the pressure the tumor size was causing.
Image onscreen:
Julie is seated in an interview chair, talking to at an interviewer off-camera.
Julie:
Within weeks, we noticed his symptoms had started to ease.
Image onscreen:
The camera follows Jacoby as he skates across the ice rink with his hockey stick. We see his father just outside the rink, leaning forward with his forearms resting on the divider. He watches Jacoby skate by and shouts encouragement. A disclaimer graphic appears along the bottom of the screen, followed by a second disclaimer graphic.
Text onscreen:
GOMEKLI may cause serious side effects, including: eye problems, heart problems, and skin problems. Tell your healthcare provider if you are pregnant or plan to become pregnant. GOMEKLI can harm your unborn baby.
Text onscreen:
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
Julie:
Throughout treatment, he experienced some skin problems including mild rashes and skin infection, but using a cream that his doctor recommended helped. He also had nausea, vomiting, diarrhea, tumor pain, intermittent headaches, and intermittent joint pain.
Image onscreen:
Julie is seated in an interview chair, talking to at an interviewer off-camera. A disclaimer graphic appears along the bottom of the screen.
Text onscreen:
These are not all of the possible side effects of GOMEKLI. Call your doctor for medical advice about side effects.
Julie:
The off weeks when he didn’t take the medication were difficult, but we worked with his health care provider to manage the side effects through hydration, rest, and over-the-counter pain relievers.
Image onscreen:
Jacoby does a slalom exercise by skating through a line of orange cones on the ice. The video briefly cuts back to Julie’s interview shot before showing Jacoby’s father using his foot to push pucks across the ice to Jacoby. Jacoby lines up in front of the goal and uses his stick to hit the pucks from his father into the goal. A disclaimer graphic appears along the bottom of the screen.
Text onscreen:
Individual results may vary.
Julie:
He also experienced a decrease in the amount of blood pumped by his heart. This did not cause symptoms and resolved without changing the dose of GOMEKLI. But Jacoby is tough, and he stuck with it. After one year of being on GOMEKLI, we got the results we wanted, met the goals of the trial, and his tumor had shrunk. Jacoby continued on GOMEKLI as part of the optional long-term follow-up phase of the trial. As part of that, he continues to get regular MRIs.
Image onscreen:
Julie is seated in an interview chair, talking to at an interviewer off-camera.
Julie:
Jacoby was only seven years old when he started treatment with GOMEKLI and there has been partial regrowth of the PN as he has continued in the study.
Image onscreen:
Jacoby and his father have left the ice and now sit down together on a bench. Jacoby takes off his helmet and talks with his father. The video briefly cuts to Julie’s interview shot before going back to Jacoby and his father sitting on the bench. Jacoby’s father affectionately ruffles Jacoby’s hair.
Julie:
His doctor has told us that this happens in some patients, and he remains on GOMEKLI treatment. I still believe that starting GOMEKLI and continuing on GOMEKLI is the right choice for us. This is just our experience and every patient’s experience is different.
Image onscreen:
Jacoby is seated in an interview chair, talking to at an interviewer off-camera. The background shows a living area with chairs, French doors leading to another room, and artwork on the walls. A name super graphic appears onscreen.
Text onscreen:
Jacoby
Living with NF1-PN and taking GOMEKLI (mirdametinib)
Jacoby is being compensated by SpringWorks Therapeutics, Inc.
Jacoby:
When I go in to talk to my doctor, he’s really down to earth.
Image onscreen:
Jacoby and his parents walk through a wooded park. Jacoby’s dad is holding a travel coffee mug. The video intermittently cuts back to Jacoby’s interview shot before continuing with the walking scene. The family continues to walk through the park and along a small lake, talking with each other and smiling. They walk over to a bench, sit down, and continue talking.
Jacoby:
He makes things really easy to understand. The way that my doctor talks to me makes me feel like this is something that I can handle for the rest of my life, so it’s easier to focus on the things I love, like hockey, football, guitar, and just life.
Julie:
It’s been incredible watching Jacoby learn to advocate for himself. He and his doctor have such a good relationship. He asked questions, he understands what his medication does, and that matters because having the right team, having doctors who listen to him, who respect him, has made all the difference.
Image onscreen:
Julie is seated in an interview chair, talking to at an interviewer off-camera.
Julie:
If I could go back and tell anything to myself when Jacoby was a baby, I would say, “Don't worry, it’s going to be okay.”
Image onscreen:
Jacoby sits in between his parents on the park bench as they continue to talk. His parents then lean in on either side of him and give him a hug. The video fades to white.
Julie:
I would tell other parents to trust your instincts. Push for answers. Surround yourself with doctors who listen and know that NF1-PN is not a roadblock. It's just a different path.
Image onscreen:
The GOMEKLI logo appears large in the center of the screen with disclaimer information in multiple lines along the bottom.
Text onscreen:
©2025 SpringWorks Therapeutics, Inc. All rights reserved. GOMEKLI is a registered trademark of SpringWorks Therapeutics, Inc. SpringWorks Therapeutics, Inc. is a healthcare company of Merck KGaA, Darmstadt, Germany. C_GOM_US_0294 11/25
We see Jacoby sitting casually on a carpeted floor, leaning back against an armchair as he strums an electric guitar that is plugged into an amplifier. He is in a living area of a home with two armchairs, a table with a frame and houseplant, and artwork on the walls. Closeup shot of Jacoby’s left hand playing chords on the neck of the guitar.
Julie:
Jacoby has always been this bright, happy kid full of energy, full of curiosity. He loves music, sports, and just being a kid.
Image onscreen:
Julie is seated in an interview chair, talking to an interviewer off-camera. The background shows a living space with an armchair, a window seat with pillows, a piano, and a side table with stacked books. A name super graphic appears onscreen.
Text onscreen:
Julie
Mother and caregiver to Jacoby living with NF1-PN and taking GOMEKLI (mirdametinib) Julie is being compensated by SpringWorks Therapeutics, Inc.
Julie:
But behind all of that, there’s this part of his story that has shaped so much of our lives – his journey with neurofibromatosis type 1 with plexiform neurofibromas or NF1-PN.
Image onscreen:
Jacoby makes an adjustment on his amplifier by turning a couple knobs. He then continues to strum his guitar. Closeup shots of his hands and face as he plays.
Julie:
His dad and I first noticed something when Jacoby was just a few months old. He had this cute coffee colored mark on his belly. At first, we didn’t think much of it and thought it might just be a birthmark, but as we started paying more attention, we noticed more of them.
Image onscreen:
Julie is seated in an interview chair, talking to at an interviewer off-camera.
Julie:
We didn’t know what they meant. I did what most new parents would do – I searched the internet. I read that Jacoby’s marks might be something called café-au-lait spots.
Image onscreen:
We see a shot of a blue-colored lava lamp, with bubbles slowly morphing inside the lamp. The video then switches back to Jacoby as he makes some adjustments on the tuning knobs on the head of his guitar. He continues to strum the guitar, then gets up, sets his guitar down on a stand, and unplugs a cable from the amplifier.
Julie:
And that’s the first time I came across neurofibromatosis type 1, or NF1. I remember reading NF1 is a genetic disorder that can cause non-cancerous tumors to grow along nerves throughout the body.
Image onscreen:
Julie is seated in an interview chair, talking to at an interviewer off-camera.
Julie:
My heart sank. No one in our family had ever faced something like this. It wasn’t something that had ever been on our radar. But my gut told me something was wrong, and I pushed for answers.
Image onscreen:
Julie is seated on a large corner couch. The wall behind has wood wainscoting and there is a round coffee table with a tray and candle in front of Julie. She holds a photo album in her lap. Jacoby walks into frame and joins her on the couch. Together, they look through the photo album and point and reminisce over family photos.
Julie:
I brought it up to our pediatrician, but she wasn’t familiar with NF1 and initially wasn’t too concerned. She did some research, made some calls, and got us connected with the NF1 clinic at the local university. The clinic took a sample of Jacoby’s blood to run tests. When the results came back, we got the official diagnosis of NF1.
Image onscreen:
Julie is seated in an interview chair, talking to at an interviewer off-camera.
Julie:
We were scared. We didn’t know what to do. The NF1 team told us all we could do was wait and see how Jacoby’s symptoms progressed.
Image onscreen:
Julie and Jacoby continue to look through the photo album.
Julie:
For a while life felt normal. Jacoby was happy, active, hitting all of his milestones. But then, around five-years-old, he started waking up in the middle of the night with intense ankle pain. Doctors told us NF1 patients often experience pain like this, and they didn’t think it was a tumor. And then one day I pressed on his abdomen, and he jolted in pain.
Image onscreen:
Julie is seated in an interview chair, talking to at an interviewer off-camera.
Julie:
I knew something was wrong. I didn’t want my son to keep experiencing this pain and discomfort. That's when I demanded imaging.
Image onscreen:
Julie and Jacoby continue to look through the photo album.
Julie:
Jacoby’s scan showed a large plexiform neurofibroma, or PN, that could not completely be removed by surgery. It had been growing all along pressing on nerves in his abdomen and groin.
Image onscreen:
Julie is seated in an interview chair, talking to at an interviewer off-camera.
Julie:
That’s when we realized that we weren’t just dealing with NF1. We were dealing with NF1-PN. Jacoby’s tumor could keep growing, causing pain, affecting his mobility. It was terrifying.
Image onscreen:
Jacoby is seated on a bench in an ice-hockey arena. He is talking to his father, who is seated on the bench next to him. We see hockey equipment on the bench next to Jacoby. Jacoby then puts on his ice skates. Closeup shot of Jacoby tying his skate laces. A disclaimer graphic appears along the bottom of the screen.
Julie:
But our NF doctor was incredible. The moment he showed us the scan he said, “There’s a clinical trial and I think Jacoby would be perfect for it.” That’s how we first heard about mirdametinib, what is now GOMEKLI.
Text onscreen:
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.
Image onscreen:
Julie is seated in an interview chair, talking to at an interviewer off-camera.
Julie:
We talked with Jacoby’s doctor about what to expect during the trial.
Image onscreen:
Closeup shot of Jacoby grabbing his gloves off the bench. We then see Jacoby putting on his helmet and mask. He then grabs his hockey stick.
Julie:
His doctor said the treatment goal was to reduce the size of his plexiform neurofibroma by at least 20%. We trusted our doctor and wanted to do whatever we could to help Jacoby.
Image onscreen:
Julie is seated in an interview chair, talking to at an interviewer off-camera.
Julie:
We decided we wanted to be in this trial.
Image onscreen:
Jacoby and his father stand up from the bench and walk along the edge of the rink. A disclaimer graphic appears along the bottom of the screen.
Text onscreen:
Please see Important Safety Information throughout and Full Prescribing Information including Patient Information.
Julie:
We hoped with the reduction of the tumor that Jacoby might get some relief from the pressure the tumor size was causing.
Image onscreen:
Julie is seated in an interview chair, talking to at an interviewer off-camera.
Julie:
Within weeks, we noticed his symptoms had started to ease.
Image onscreen:
The camera follows Jacoby as he skates across the ice rink with his hockey stick. We see his father just outside the rink, leaning forward with his forearms resting on the divider. He watches Jacoby skate by and shouts encouragement. A disclaimer graphic appears along the bottom of the screen, followed by a second disclaimer graphic.
Text onscreen:
GOMEKLI may cause serious side effects, including: eye problems, heart problems, and skin problems. Tell your healthcare provider if you are pregnant or plan to become pregnant. GOMEKLI can harm your unborn baby.
Text onscreen:
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
Julie:
Throughout treatment, he experienced some skin problems including mild rashes and skin infection, but using a cream that his doctor recommended helped. He also had nausea, vomiting, diarrhea, tumor pain, intermittent headaches, and intermittent joint pain.
Image onscreen:
Julie is seated in an interview chair, talking to at an interviewer off-camera. A disclaimer graphic appears along the bottom of the screen.
Text onscreen:
These are not all of the possible side effects of GOMEKLI. Call your doctor for medical advice about side effects.
Julie:
The off weeks when he didn’t take the medication were difficult, but we worked with his health care provider to manage the side effects through hydration, rest, and over-the-counter pain relievers.
Image onscreen:
Jacoby does a slalom exercise by skating through a line of orange cones on the ice. The video briefly cuts back to Julie’s interview shot before showing Jacoby’s father using his foot to push pucks across the ice to Jacoby. Jacoby lines up in front of the goal and uses his stick to hit the pucks from his father into the goal. A disclaimer graphic appears along the bottom of the screen.
Text onscreen:
Individual results may vary.
Julie:
He also experienced a decrease in the amount of blood pumped by his heart. This did not cause symptoms and resolved without changing the dose of GOMEKLI. But Jacoby is tough, and he stuck with it. After one year of being on GOMEKLI, we got the results we wanted, met the goals of the trial, and his tumor had shrunk. Jacoby continued on GOMEKLI as part of the optional long-term follow-up phase of the trial. As part of that, he continues to get regular MRIs.
Image onscreen:
Julie is seated in an interview chair, talking to at an interviewer off-camera.
Julie:
Jacoby was only seven years old when he started treatment with GOMEKLI and there has been partial regrowth of the PN as he has continued in the study.
Image onscreen:
Jacoby and his father have left the ice and now sit down together on a bench. Jacoby takes off his helmet and talks with his father. The video briefly cuts to Julie’s interview shot before going back to Jacoby and his father sitting on the bench. Jacoby’s father affectionately ruffles Jacoby’s hair.
Julie:
His doctor has told us that this happens in some patients, and he remains on GOMEKLI treatment. I still believe that starting GOMEKLI and continuing on GOMEKLI is the right choice for us. This is just our experience and every patient’s experience is different.
Image onscreen:
Jacoby is seated in an interview chair, talking to at an interviewer off-camera. The background shows a living area with chairs, French doors leading to another room, and artwork on the walls. A name super graphic appears onscreen.
Text onscreen:
Jacoby
Living with NF1-PN and taking GOMEKLI (mirdametinib)
Jacoby is being compensated by SpringWorks Therapeutics, Inc.
Jacoby:
When I go in to talk to my doctor, he’s really down to earth.
Image onscreen:
Jacoby and his parents walk through a wooded park. Jacoby’s dad is holding a travel coffee mug. The video intermittently cuts back to Jacoby’s interview shot before continuing with the walking scene. The family continues to walk through the park and along a small lake, talking with each other and smiling. They walk over to a bench, sit down, and continue talking.
Jacoby:
He makes things really easy to understand. The way that my doctor talks to me makes me feel like this is something that I can handle for the rest of my life, so it’s easier to focus on the things I love, like hockey, football, guitar, and just life.
Julie:
It’s been incredible watching Jacoby learn to advocate for himself. He and his doctor have such a good relationship. He asked questions, he understands what his medication does, and that matters because having the right team, having doctors who listen to him, who respect him, has made all the difference.
Image onscreen:
Julie is seated in an interview chair, talking to at an interviewer off-camera.
Julie:
If I could go back and tell anything to myself when Jacoby was a baby, I would say, “Don't worry, it’s going to be okay.”
Image onscreen:
Jacoby sits in between his parents on the park bench as they continue to talk. His parents then lean in on either side of him and give him a hug. The video fades to white.
Julie:
I would tell other parents to trust your instincts. Push for answers. Surround yourself with doctors who listen and know that NF1-PN is not a roadblock. It's just a different path.
Image onscreen:
The GOMEKLI logo appears large in the center of the screen with disclaimer information in multiple lines along the bottom.
Text onscreen:
©2025 SpringWorks Therapeutics, Inc. All rights reserved. GOMEKLI is a registered trademark of SpringWorks Therapeutics, Inc. SpringWorks Therapeutics, Inc. is a healthcare company of Merck KGaA, Darmstadt, Germany. C_GOM_US_0294 11/25
Hear Julie and her son Jacoby talk about his experience on GOMEKLI treatment.
My doctor makes me feel like this is something I can handle for the rest of my life, so it’s easier to focus on the things I love.
– Jacoby, GOMEKLI Patient Ambassador
Individual results may vary.
Ready to share your experience on GOMEKLI?
The GOMEKLI Patient Ambassador Program offers a meaningful opportunity to raise awareness and share your story—whether you’re taking GOMEKLI or caring for someone on treatment.‡
Call or email today to share your story.
‡
Qualified candidates must be 18 years of age or older to participate.
In children/adolescents who benefited from treatment, plexiforms started to shrink within months of taking GOMEKLI
In children/adolescents who benefited from treatment, plexiforms began to shrink by at least 20% within 4 to 19 months of starting GOMEKLI. The median (middle) amount of time it took for plexiforms to begin shrinking was 7.9 months.
The GOMEKLI clinical trial was about 2 years long. After that,
85% of eligible children/adolescents (28 out of 33) chose to keep taking GOMEKLI in an optional long-term follow-up phase of the study.
Watch this video of GOMEKLI study investigator Dr. Hirbe walking viewers through the results of the clinical study.
Possible side effects with GOMEKLI (mirdametinib)
GOMEKLI may cause serious side effects, including:
Eye problems
Tell your healthcare provider right away if you get any of the following signs or symptoms of eye problems:
- Blurred vision, loss of vision, or other changes to your vision
Heart problems
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
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
In children and adolescents, the most common ones were:
- Diarrhea
- Muscle, joint, and bone pain
- Stomach (abdominal) pain
- Vomiting
- Headache
- Nausea
- Skin redness, swelling, or pain around the fingernails or toenails
The most common severe abnormal blood test results in children and adolescents involved decreased white blood cell counts and an increased enzyme called creatine phosphokinase (CPK).
These are not all 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.
Most children/adolescents taking GOMEKLI did not have to stop treatment due to a side effect
had to stop treatment due to a side effect
Your healthcare provider may interrupt, reduce, or permanently stop GOMEKLI treatment if you experience certain side effects.
Managing side effects
Managing side effects
Learn how to work with your care team to manage the possible side effects of GOMEKLI.
Image onscreen:
Petal shape from GOMEKLI logo appears onscreen, spinning as if the video is loading. Petal shape comes to a stop as full GOMEKLI logo fades in.
Image onscreen:
GOMEKLI logo shrinks and moves to top center of the frame. Indication statement appears.
Text onscreen and voice-over:
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 see Important Safety Information later in this video and Patient Information available on GOMEKLI.com.
Image onscreen:
GOMEKLI logo fades and a flag with the logo in the top right corner of the frame appears, where it remains for the entirety of the video. Title of video fades in.
Text onscreen:
Managing possible side effects of GOMEKLI
Voice-over:
In this video, we’ll talk about how to work with your care team to manage common side effects and offer tips that may help throughout treatment.
Image onscreen:
Word bubbles pop up around a woman as the different side effects are introduced. The woman shrinks and moves to the bottom left of the screen as the child comes into view. The word bubble bubbles pop up around him as pediatric symptoms are introduced. The shot pans to the woman sitting across from her doctor.
Voice-over:
The most common side effects with GOMEKLI are diarrhea, nausea, muscle, joint, and bone pain, and vomiting. Some adults may also experience tiredness, while some children may experience headache, stomach pain, and skin redness, swelling, or pain around the fingernails and toenails.
GOMEKLI can cause other side effects, including some serious side effects which we will go over in this video.
Most of the side effects associated with GOMEKLI have been shown to start early in treatment, but it’s good to know that there are things your care team can do to help address them.
Image onscreen:
The camera zooms in to the woman’s arm with a rash. Different rash types appear in word bubbles around her as they are introduced.
Text onscreen:
One of the most common side effects with GOMEKLI is rash.
Voice-over:
In the single-arm clinical study of GOMEKLI, one of the most common side effects seen among both adults and children was rash, which can be severe. This could be a flat skin rash, raised bumps on the skin, skin bumps that look like acne, skin redness, itchy rash, or peeling skin.
Image onscreen:
The woman is shown with her doctor as she examines her rash. The screen splits. On the left the woman in a bathtub. On the right, the woman is applying lotion to her arm.
Text onscreen:
This information is not intended to replace the advice of your care team or other healthcare provider.
Voice-over:
There are things you can do to help address skin-related side effects. These include taking daily baths and using mild cleansers and skin moisturizers at least twice a day to prevent dryness. You may also consider avoiding products that could dry out or irritate your skin.
Image onscreen:
A doctor types up a prescription for her patient.
Text onscreen:
Tell your care team if you develop any signs or symptoms of skin problems.
Voice-over:
Your care team may also provide medication to help with the rash or refer you to a dermatologist for additional care.
Image onscreen:
A woman holds her stomach in discomfort. Different types of gastrointestinal appear in word bubbles around her as they are introduced.
Text onscreen:
Diarrhea, nausea, vomiting, and abdominal pain are also common side effects.
Voice-over:
Gastrointestinal side effects, such as diarrhea, nausea, vomiting, and abdominal pain, are also common in people who take GOMEKLI.
Image onscreen:
Food icons of french fries, hot peppers, and potato chips appear on screen in large white circles and lines going through them. They disappear and a glass appears onscreen. The shot pans to a pharmacy where the woman is looking at different medications.
Text onscreen:
This information is not intended to replace the advice of your care team or other healthcare provider.
Voice-over:
There are certain tips you can keep in mind to address stomach-related side effects, such as avoiding fried, fatty, or spicy foods, and increasing your fluid intake. If you continue to experience issues, consult your care team for further treatment options.
Image onscreen:
The woman is shown speaking with her doctor. The doctor nods and takes notes.
Text onscreen:
Take GOMEKLI as directed by your healthcare provider.
Voice-over:
Depending on what you’re experiencing, your care team may interrupt, reduce, or permanently stop your GOMEKLI treatment. It is important that you take GOMEKLI as directed by your healthcare provider.
Image onscreen:
The woman nods her head as she continues speaking with her doctor. The serious side effects pop up in word bubbles around her. The doctor then begins to examine her eyes.
Text onscreen:
GOMEKLI can cause eye and heart problems that can be serious.
Voice-over:
Because GOMEKLI can cause serious side effects, including eye problems and heart problems, your care team will regularly assess your eyes and heart before and throughout treatment. Before taking GOMEKLI, make sure to tell your healthcare provider if you have any eye or heart problems.
Image onscreen:
The doctor and woman discuss her lab results.
Text onscreen:
Your care team will conduct blood tests.
Voice-over:
Your care team will also want to run routine lab work, including blood tests. These assessments are important because they help your care team see if anything is going on that needs to be addressed.
Image onscreen:
The woman checks her phone. The camera zooms in on her screen, where it shows her scrolling through her contact list.
Text onscreen:
Regular check-ins with your care team are extremely important.
Voice-over:
That’s why it’s vital to stay on top of appointments.
Image onscreen:
The woman clicks on the contact for her care team. The screen splits: On the left the woman has the phone to her ear and on the right a doctor also has a phone to her ear.
Text onscreen:
Tell your care team immediately if you notice anything out of the ordinary.
Voice-over:
But remember, if you’re experiencing anything out of the ordinary, you shouldn’t wait until your next appointment to let your care team know. The key to staying on top of side effects is being proactive, open, and honest with your care team. They won’t know how to help you if you don’t speak up!
Image onscreen:
The GOMEKLI treatment journal appears. The pages turn so viewers can see what it looks like and how it works. We fade to the woman sitting at a desk reading through the treatment journal.
Text onscreen:
Use the treatment journal to track your appointments, how you’re feeling, and more.
Voice-over:
The GOMEKLI treatment journal can make things easier. It includes weekly templates so you can record how you’re feeling, jot down general notes, and save any questions you have for your care team. Once you start GOMEKLI and are enrolled in SpringWorks CareConnections, the journal will be sent to you as part of your GOMEKLI starter kit. You can also find it on GOMEKLI.com.
Image onscreen:
The treatment journal goes away and is replaced with a laptop. On the laptop screen is GOMEKLI.com resource page, which is being scrolled through.
Text onscreen:
Find more helpful resources on GOMEKLI.com.
Voice-over:
GOMEKLI.com also has other helpful resources in case you have questions about treatment.
Image onscreen:
The woman continues to explore GOMEKLI.com and makes her way to the SpringWorks CareConnections® page.
Text onscreen:
The SpringWorks CareConnections® Patient Support Program is not intended to take the place of your healthcare provider, and our team of Nurse Advocates cannot provide medical or clinical advice.
Voice-over:
Additionally, once you’re enrolled in SpringWorks CareConnections, our team of Nurse Advocates will be available to provide support and help you stay on track with treatment.
Image onscreen:
QUICK CUTS: Woman takes notes in her journal. She calls her doctor. Doctor is shown speaking on the phone.
Voice-over:
Managing side effects is a team effort involving both you and your care team. Keeping an open line of communication is key to ensuring you have the best experience possible with GOMEKLI. If you have any questions or concerns, please reach out to your healthcare provider. Keep watching for additional important information on side effects with GOMEKLI.
Text onscreen and voice-over:
Important Safety Information
Before taking GOMEKLI, tell your healthcare provider about all of your medical conditions, including if you:
How should I take GOMEKLI?
What are the possible side effects of GOMEKLI?
GOMEKLI may cause serious side effects, including:
The most common side effects of GOMEKLI in adults include:
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:
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 see full Prescribing Information, including Patient Information and Instructions for Use, available on GOMEKLI.com.
Text onscreen:
Thank you!
©2025 SpringWorks Therapeutics, Inc. All rights reserved. GOMEKLI and SpringWorks Therapeutics are registered trademarks of SpringWorks Therapeutics, Inc. C_GOM_US_0432 8/25
Voice-over:
Thank you for watching.
Petal shape from GOMEKLI logo appears onscreen, spinning as if the video is loading. Petal shape comes to a stop as full GOMEKLI logo fades in.
Image onscreen:
GOMEKLI logo shrinks and moves to top center of the frame. Indication statement appears.
Text onscreen and voice-over:
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 see Important Safety Information later in this video and Patient Information available on GOMEKLI.com.
Image onscreen:
GOMEKLI logo fades and a flag with the logo in the top right corner of the frame appears, where it remains for the entirety of the video. Title of video fades in.
Text onscreen:
Managing possible side effects of GOMEKLI
Voice-over:
In this video, we’ll talk about how to work with your care team to manage common side effects and offer tips that may help throughout treatment.
Image onscreen:
Word bubbles pop up around a woman as the different side effects are introduced. The woman shrinks and moves to the bottom left of the screen as the child comes into view. The word bubble bubbles pop up around him as pediatric symptoms are introduced. The shot pans to the woman sitting across from her doctor.
Voice-over:
The most common side effects with GOMEKLI are diarrhea, nausea, muscle, joint, and bone pain, and vomiting. Some adults may also experience tiredness, while some children may experience headache, stomach pain, and skin redness, swelling, or pain around the fingernails and toenails.
GOMEKLI can cause other side effects, including some serious side effects which we will go over in this video.
Most of the side effects associated with GOMEKLI have been shown to start early in treatment, but it’s good to know that there are things your care team can do to help address them.
Image onscreen:
The camera zooms in to the woman’s arm with a rash. Different rash types appear in word bubbles around her as they are introduced.
Text onscreen:
One of the most common side effects with GOMEKLI is rash.
Voice-over:
In the single-arm clinical study of GOMEKLI, one of the most common side effects seen among both adults and children was rash, which can be severe. This could be a flat skin rash, raised bumps on the skin, skin bumps that look like acne, skin redness, itchy rash, or peeling skin.
Image onscreen:
The woman is shown with her doctor as she examines her rash. The screen splits. On the left the woman in a bathtub. On the right, the woman is applying lotion to her arm.
Text onscreen:
This information is not intended to replace the advice of your care team or other healthcare provider.
Voice-over:
There are things you can do to help address skin-related side effects. These include taking daily baths and using mild cleansers and skin moisturizers at least twice a day to prevent dryness. You may also consider avoiding products that could dry out or irritate your skin.
Image onscreen:
A doctor types up a prescription for her patient.
Text onscreen:
Tell your care team if you develop any signs or symptoms of skin problems.
Voice-over:
Your care team may also provide medication to help with the rash or refer you to a dermatologist for additional care.
Image onscreen:
A woman holds her stomach in discomfort. Different types of gastrointestinal appear in word bubbles around her as they are introduced.
Text onscreen:
Diarrhea, nausea, vomiting, and abdominal pain are also common side effects.
Voice-over:
Gastrointestinal side effects, such as diarrhea, nausea, vomiting, and abdominal pain, are also common in people who take GOMEKLI.
Image onscreen:
Food icons of french fries, hot peppers, and potato chips appear on screen in large white circles and lines going through them. They disappear and a glass appears onscreen. The shot pans to a pharmacy where the woman is looking at different medications.
Text onscreen:
This information is not intended to replace the advice of your care team or other healthcare provider.
Voice-over:
There are certain tips you can keep in mind to address stomach-related side effects, such as avoiding fried, fatty, or spicy foods, and increasing your fluid intake. If you continue to experience issues, consult your care team for further treatment options.
Image onscreen:
The woman is shown speaking with her doctor. The doctor nods and takes notes.
Text onscreen:
Take GOMEKLI as directed by your healthcare provider.
Voice-over:
Depending on what you’re experiencing, your care team may interrupt, reduce, or permanently stop your GOMEKLI treatment. It is important that you take GOMEKLI as directed by your healthcare provider.
Image onscreen:
The woman nods her head as she continues speaking with her doctor. The serious side effects pop up in word bubbles around her. The doctor then begins to examine her eyes.
Text onscreen:
GOMEKLI can cause eye and heart problems that can be serious.
Voice-over:
Because GOMEKLI can cause serious side effects, including eye problems and heart problems, your care team will regularly assess your eyes and heart before and throughout treatment. Before taking GOMEKLI, make sure to tell your healthcare provider if you have any eye or heart problems.
Image onscreen:
The doctor and woman discuss her lab results.
Text onscreen:
Your care team will conduct blood tests.
Voice-over:
Your care team will also want to run routine lab work, including blood tests. These assessments are important because they help your care team see if anything is going on that needs to be addressed.
Image onscreen:
The woman checks her phone. The camera zooms in on her screen, where it shows her scrolling through her contact list.
Text onscreen:
Regular check-ins with your care team are extremely important.
Voice-over:
That’s why it’s vital to stay on top of appointments.
Image onscreen:
The woman clicks on the contact for her care team. The screen splits: On the left the woman has the phone to her ear and on the right a doctor also has a phone to her ear.
Text onscreen:
Tell your care team immediately if you notice anything out of the ordinary.
Voice-over:
But remember, if you’re experiencing anything out of the ordinary, you shouldn’t wait until your next appointment to let your care team know. The key to staying on top of side effects is being proactive, open, and honest with your care team. They won’t know how to help you if you don’t speak up!
Image onscreen:
The GOMEKLI treatment journal appears. The pages turn so viewers can see what it looks like and how it works. We fade to the woman sitting at a desk reading through the treatment journal.
Text onscreen:
Use the treatment journal to track your appointments, how you’re feeling, and more.
Voice-over:
The GOMEKLI treatment journal can make things easier. It includes weekly templates so you can record how you’re feeling, jot down general notes, and save any questions you have for your care team. Once you start GOMEKLI and are enrolled in SpringWorks CareConnections, the journal will be sent to you as part of your GOMEKLI starter kit. You can also find it on GOMEKLI.com.
Image onscreen:
The treatment journal goes away and is replaced with a laptop. On the laptop screen is GOMEKLI.com resource page, which is being scrolled through.
Text onscreen:
Find more helpful resources on GOMEKLI.com.
Voice-over:
GOMEKLI.com also has other helpful resources in case you have questions about treatment.
Image onscreen:
The woman continues to explore GOMEKLI.com and makes her way to the SpringWorks CareConnections® page.
Text onscreen:
The SpringWorks CareConnections® Patient Support Program is not intended to take the place of your healthcare provider, and our team of Nurse Advocates cannot provide medical or clinical advice.
Voice-over:
Additionally, once you’re enrolled in SpringWorks CareConnections, our team of Nurse Advocates will be available to provide support and help you stay on track with treatment.
Image onscreen:
QUICK CUTS: Woman takes notes in her journal. She calls her doctor. Doctor is shown speaking on the phone.
Voice-over:
Managing side effects is a team effort involving both you and your care team. Keeping an open line of communication is key to ensuring you have the best experience possible with GOMEKLI. If you have any questions or concerns, please reach out to your healthcare provider. Keep watching for additional important information on side effects with GOMEKLI.
Text onscreen and voice-over:
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
- 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.
Females who are able to become pregnant: -
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.
Please see full Prescribing Information, including Patient Information and Instructions for Use, available on GOMEKLI.com.
Text onscreen:
Thank you!
©2025 SpringWorks Therapeutics, Inc. All rights reserved. GOMEKLI and SpringWorks Therapeutics are registered trademarks of SpringWorks Therapeutics, Inc. C_GOM_US_0432 8/25
Voice-over:
Thank you for watching.
Learn how to work with your care team to manage the possible side effects of GOMEKLI.
My son Jacoby has experienced headaches and nausea during his treatment-free weeks. To get ahead of this, we give him an over-the-counter pain reliever 1 or 2 days after his off week starts. It’s helped.
– Julie, GOMEKLI Caregiver Ambassador
Individual results may vary.
Find the care you need
Use the doctor finder tool to locate a healthcare provider near you who has experience with NF1.
Is GOMEKLI right for me?
This guide can help you and your healthcare provider understand if GOMEKLI could be right for you.
Dosing that fits your needs
Available in capsules or tablets for oral suspension that give you the option to take GOMEKLI as a liquid.
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.