Holmes, lives with pLGG, and Clare, Holmes' mom
Safety and
side effects
In the FIREFLY-1 study, most side effects reported in children were mild to moderate, and managed
89%
of children did not stop taking OJEMDA due to side effects
(122 out of 137)
The side effects that caused 11% of children (15 out of 137) to stop taking OJEMDA were:
- Bleeding from the tumor (4 out of 137)
- Slowing of growth (4 out of 137)
If your child experiences any serious side effects during treatment with OJEMDA, your child’s doctor may decrease dosage.
The FIREFLY-1 main phase concluded in 2024. Patients are currently being followed for longer-term observation.
Managing side effects, together as a family
Hear from caregivers about how they worked with their care teams to manage side effects for their children taking OJEMDA
ON SCREEN: REAL FAMILIES. REAL EXPERIENCES. MANAGING SIDE EFFECTS OF OJEMDA™ (tovorafenib)
REEBA: We always talk about how Myla is probably going to be the toughest kid on the street because of everything she's gone through.
ON SCREEN: REEBA mother of Myla who was treated with OJEMDA for 25 months
REEBA: But I also think that's why she's so laid back about things, is she's gone through so much and very little fazes her.
AARON: Olive had been through multiple different treatment options
ON SCREEN: TRISTA & AARON parents of Olive who was treated with OJEMDA for 25 months
AARON: over a span of years, and sometimes it was a lot to manage.
TRISTA: But she handled it like a champ.
MANDY: Gabby is her own person in every sense of the word. And this time with this treatment, that resilience has sort of carried us through.
ON SCREEN: MANDY mother of Gabby who was treated with OJEMDA for 25 months
ON SCREEN: Experience with Side Effects
REEBA: It was a little terrifying to know that your child was going on a trial and you weren't sure exactly what was going to happen. But what they could tell us was information that we could kind of wrap our heads around and we felt comfortable with it. So we decided to trust the care team once more and decided to go ahead and give the trial for OJEMDA a shot.
ON SCREEN: What is OJEMDA™ (tovorafenib)? OJEMDA is a prescription medicine used to treat certain types of brain tumors (cancers) called gliomas in people 6 months and older:
• that is a pediatric low-grade glioma (LGG), and
• that has come back after previous treatment or has not responded to previous treatment, and
• that has a certain type of abnormal "BRAF" gene.
• that is a pediatric low-grade glioma (LGG), and
• that has come back after previous treatment or has not responded to previous treatment, and
• that has a certain type of abnormal "BRAF" gene.
Please see full Important Safety Information at the end of this video.
MANDY: Gabby experienced rash, nausea, vomiting, fatigue, and perhaps most noticeably, hair color change.
REEBA: Myla did have some side effects while she was on OJEMDA. She did have a rash that would cover her body and her face occasionally. She had sensitivity to sunlight. She did have slowed growth. She would get nauseous and would throw up a lot when she was really hot.
ON SCREEN: OJEMDA may cause serious side effects, including bleeding problems, skin reactions, including sensitivity to sunlight (photosensitivity), liver problems and slowing of growth (height).
AARON: I think it was a couple weeks in when she started to get the rash, but we stayed in really close contact with the care team. We had a number of different creams and ointments that she needed.
ON SCREEN: Rash typically appeared 2 weeks after the first dose and up to 16 months after.
TRISTA: We did a lot of bleach baths, which sounds wild, but it's just a bath with a little bit of bleach in it.
AARON: She was not a fan.
ON SCREEN: OJEMDA was generally well tolerated in the clinical trial
TRISTA: Not a fan, especially not a fan of doing all the, like, creams afterward. So we had to be creative about how to get her, like, excited about the baths on really hard days. ON SCREEN: 7% of children (9 out of 137) stopped taking OJEMDA due to side effects
TRISTA: One time, I had like a whole glow, set up in the bath with funky music and balloons and glow sticks and stuff. We also, you know, had times where we turned it into a spa with aromatherapy and just relaxing.
REEBA: One of the side effects that I mentioned earlier is that Myla would get nauseous and she would throw up. This would especially happen when she was hot, when she would get overheated and she'd been in the sun for too long. But it wasn't always that simple. I appreciate my daughters both so much, but my daughter Ava, Myla’s younger sister, who's about a year and a half younger than her, would be kind of my alarm system. We would be sitting on the couch together,
ON SCREEN:
The most common side effects of OJEMDA include:
• rash
• hair color changes
• Feeling tired
• viral infection
• vomiting
• headache
• fever
• dry skin
• constipation
• nausea
• acne
• upper respiratory tract infection
REEBA: and before I could even notice that it was happening, Ava somehow had this sixth sense that Myla was starting to feel sick, and she would look at me and say, “Mommy, go get the throw up bucket. Myla is about to throw up.” Right? So it was horrible, right? But it was amazing to watch Ava be that caregiver for Myla and be so attuned to what was happening with her when she was starting to get sick.
AARON: Olive, she also had pretty severe sun sensitivity.
TRISTA: Yeah. Sometimes it took a little extra prodding, but we tried to get her out and about by taking precautions. We even went to Hawaii. We did a lot of fun stuff. So, it was something that was tolerable for our family.
AARON: The side effect that is clearly the most notable would be the white hair.
TRISTA: And white eyebrows and eyelashes.
REEBA: The hair color change was, was an impact. It didn't bother her as much. I will say her eyebrows and her eyelashes did bother her. She felt a little strange about those. And as a mom, you always want to protect your kids. And so I was so worried that, you know, she was in fourth grade going into fifth grade, it was her last two years of elementary school. And I was so worried the kids would make fun of her because her hair color was changing. I will tell you, it was almost the exact opposite. Kids thought it was so cool. She always looked gorgeous no matter what. She's so sweet from the inside, and that just radiates.
AARON: Olive really leaned in on the white hair.
TRISTA: If people would suggest that she could dye it fun colors or something like that, she was like, no, it’s awesome.
MANDY: Gabby embraced this change of hair color, and she took that change of hair color just like anything else, and she just rolled with it. We called her a superhero.
REEBA: One of the lesser known side effects while Myla was on OJEMDA was slowed growth. It's really hard to notice that, right, when you're with your child every day, you don't really notice that their growth has slowed. But throughout her treatment, they did do measurements on her height. And, although we didn't really pay attention to it, we noticed that it was, it was slowing. After about three months of stopping treatment, we noticed that Myla was growing again. And at about nine months, she was catching up.
TRISTA: It meant so much for us for Olive to be able to take the medication once a week, at home, with or without food.
ON SCREEN:
OJEMDA can be taken:
• once weekly
• at home
• with or without food
• as tablets or liquid
REEBA: She had five really large pills that she had to take once a week, but the once a week piece was really important to us because we didn't have to shuttle back and forth between the hospital anymore.
ON SCREEN: Message to Other Parents
AARON: When you’re dealing with a brain tumor, doing nothing is often not an option.
MANDY: When we came along this with the opportunity to try an investigational drug, we felt like we had to take that chance. And part of why we took that chance is because we knew that other families along the way had taken this same chance so that the drugs Gabby had already taken had already been through the investigational process, were already FDA now approved because somebody else said yes.
AARON: Honestly, just felt really good knowing that we were going to be helping move pediatric brain tumor research forward.
REEBA: If I were to talk to another family who is considering OJEMDA, there's a few things that I would tell you to consider. Number one: Trust yourself and understand that you know your child better than anybody else. Make sure that you are asking the questions that you need answers to so that you feel safe and you feel educated and you can advocate for your child.
MANDY: You have a voice in this process. And don't be afraid to use that voice to share your concerns or things you may have come across and share with your care team so that you together can make those decisions.
REEBA: My number one piece of advice is you are going to be your child's greatest advocate.
“Side effects can happen with any medication. It is important to discuss any symptoms your child may experience while taking OJEMDA so that your doctor can advise on the appropriate course of action.”
—Dr Peter Manley, Vice President, Clinical Development, at Day One Biopharmaceuticals
What are the possible side effects?
OJEMDA may cause serious side effects, including
Bleeding problems,
such as nose bleeds or bleeding from the tumor
Call your child’s doctor immediately if they have any symptoms, including:
- Headache, dizziness, or feeling weak
- Coughing up blood or blood clots
- Vomiting blood or vomit looks like coffee grounds
- Red or black stools that look like tar
Skin reactions, including sensitivity to sunlight (photosensitivity)
Call your child’s doctor if your child gets new or worsening skin reactions, including:
- Rash
- Bumps
- Acne
- Peeling, redness, or irritation
- Blisters
Liver problems
Your child’s doctor will do blood tests to check your child’s liver function before and during treatment with OJEMDA. Call your child’s doctor right away if your child gets any of the following symptoms:
- Yellowing of skin or eyes
- Dark or brown (tea-colored) urine
- Nausea or vomiting
- Loss of appetite
- Feeling tired
- Bruising
- Bleeding
- Pain in the upper right stomach area
Slowing of growth (height)
- Your child’s growth will be checked routinely during treatment with OJEMDA
- Children's growth improved after pausing or stopping OJEMDA
Other safety considerations
OJEMDA may cause fertility problems in female and male children, which can affect their ability to have children. If you have concerns about your child’s fertility, you should discuss their family planning goals with their care team. Remember, your care team is there to support you every step of the way.
What are the most common side effects?
The 137 children in the clinical study mostly experienced mild to moderate side effects while taking OJEMDA. Seeing and hearing what other families have gone through may be helpful. But no one knows your child like you, so reach out to your care team right away if they experience any side effects, and discuss ways to manage them.
Side effect
Out of 137 children, what percent experienced it?
These data include patients who completed up to 2 years of treatment with OJEMDA in the clinical trial, and additional analyses were done. These data are not reflected in the Prescribing Information.
These are not all the possible side effects with OJEMDA. Talk to your child’s care team for medical advice about possible side effects.
You may report side effects to the FDA at 1-800-FDA-1088.
You may also report side effects to Day One Biopharmaceuticals at
