Frequently asked questions

OJEMDA is a prescription medicine used to treat certain types of brain tumors (cancers) called gliomas in patients 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.

OJEMDA is a prescription oral medication for children 6 months or older with pediatric low-grade glioma that has returned or did not respond to treatment and who have a certain type of abnormal BRAF gene.

Your healthcare provider will perform a test to make sure that OJEMDA is right for you.

It is not known if OJEMDA is safe and effective in children younger than 6 months of age.

No, OJEMDA is a targeted therapy. Most types of targeted therapies are designed to block the signals that fuel growth of tumor cells.

OJEMDA is designed to block tumor growth caused by changes in the BRAF gene, most commonly BRAF fusions and BRAF V600E mutations. A genomic test can help determine if your child has a change in the BRAF gene in their tumor.

Pediatric low-grade gliomas (pLGG) are the most common type of brain tumors diagnosed in children. These slow-growing tumors are found in different areas of the brain and spinal cord.

pLGG can impact a child’s health in many ways. Depending on the tumor’s size and location in the brain, symptoms can include vision problems and motor dysfunction.

Genomic testing plays a crucial role in tailoring treatment plans. The choice of treatment may depend on factors such as the specific characteristics of the tumor, previous treatments, and the overall health of the patient.

Balancing treatment decisions with the impact on the child’s life is essential. Discussions with your child’s care team should include considerations for potential results, side effects, and the impact on their activities. Regular imaging studies and clinical evaluations help track the tumor’s response to treatment and detect any potential signs of recurrence.

Learn more about pLGG

OJEMDA was studied in a clinical trial called FIREFLY-1. The study looked at whether OJEMDA is a safe and effective way to keep tumors from growing in children with BRAF fusions or rearrangements or BRAF V600E mutations, whose tumors came back or did not respond to a previous treatment like chemotherapy or a targeted treatment.

OJEMDA was not compared to other treatments.

The study is still ongoing and full trial results will be reviewed by the end of 2024.

The clinical trial involved 137 children, and the goals of the study were to understand the efficacy and safety.

For information about the clinical trial results, download the Guide for Considering OJEMDA

Meaningful tumor shrinkage was seen in the clinical trial, FIREFLY-1:

• 51% (39 out of 76 children) saw their pLGG tumors shrink by at least 25%
  • 37% (28 out of 76 children) had tumor shrinkage of 50% or more
  • No child had their tumor completely disappear

Additional results:

• 57% (43 out of 76 children) experienced tumor shrinkage, or their tumor size remained stable for 12 months or more
• 82% (62 out of 76 children) experienced tumor shrinkage, or their tumor size remained stable for 3 months or more
  • Stability was defined as the tumor not growing or shrinking more than 25%

These data were collected during the clinical study, and additional analysis was done. These results are not in the OJEMDA Prescribing Information.

The most common side effects of OJEMDA include

• Rash
• Hair color changes
• Tiredness
• Viral infection
• Vomiting
• Headache
• Fever
• Dry skin
• Constipation
• Nausea
• Acne
• Upper respiratory tract infection

For information about clinical trial results, download the Guide for Considering OJEMDA

Results were measured with MRI scans every 3 months. 46% of children who experienced tumor shrinkage (18 out of 39) saw it on an MRI scan at 3 months after starting treatment, and 77% of children (30 out of 39) saw it on an MRI scan at 6 months.

Learn more about results with OJEMDA

This data was collected during the clinical study, and additional analysis was done. These results are not in the OJEMDA Prescribing Information.

At the time of review, half of the children who saw tumor shrinkage saw it for 13.8 months.

Learn more about results with OJEMDA

In the clinical study, children mostly experienced mild to moderate side effects. When the results of the FIREFLY-1 trial were studied:

• 74% of children were still taking OJEMDA (102 out of 137 children)
• 93% of children did not stop taking OJEMDA due to side effects (128 out of 137 children). The main side effects that caused 7% of children (9 out of 137) to stop taking OJEMDA were:
  • Bleeding from the tumor (3 out of 137)
  • Slowing of growth (2 out of 137)

If your child experiences any serious side effects during treatment with OJEMDA, your child’s doctor may decrease dosage, pause treatment, or stop treatment.

The most common side effects of OJEMDA include

• Rash
• Hair color changes
• Tiredness
• Viral infection
• Vomiting
• Headache
• Fever
• Dry skin
• Constipation
• Nausea
• Acne
• Upper respiratory tract infection

For information about clinical trial results, download the Guide for Considering OJEMDA

OJEMDA has not been compared in a head-to-head study against other treatments for pLGG.

Your child’s duration of therapy will be assessed by your child’s doctor.

Learn more about taking OJEMDA

The FIREFLY-1 study of OJEMDA will conclude in 2024. The full clinical trial data set will be released after that data has been reviewed.

OJEMDA may cause serious side effects, including

• Bleeding problems (hemorrhage) are common and can also be serious. Tell your healthcare provider if you develop any signs or symptoms of bleeding, including:
  • Headache, dizziness or feeling weak
  • Coughing up blood or blood clots
  • Vomit blood or your vomit looks like coffee grounds
  • Red or black stools that look like tar
• Skin reactions, including sensitivity to sunlight (photosensitivity). OJEMDA can cause skin reactions that can become severe. Tell your healthcare provider if you get new or worsening skin reactions, including:
  • Rash
  • Bumps
  • Acne
  • Peeling, redness, or irritation
  • Blisters
• Liver problems. Your healthcare provider will do blood tests to check your child’s liver function before and during treatment with OJEMDA. Tell your healthcare provider right away if your child develops any of the following symptoms:
  • Yellowing of your skin or your eyes
  • Dark or brown (tea-colored) urine
  • Nausea or vomiting
  • Loss of appetite
  • Tiredness
  • Bruising
  • Bleeding
  • Pain in your upper right stomach area
• Slowed growth in children. Growth will be checked routinely during treatment with OJEMDA.

These are not all the possible side effects of OJEMDA. Call your child’s care team for medical advice about possible side effects. You may report side effects to FDA at 1-800-FDA-1088. You may also report side effects to Day One Biopharmaceuticals at 1-855-DAY1-BIO (1-855-329-1246).

The most common side effects of OJEMDA include

• Rash
• Hair color changes
• Tiredness
• Viral infection
• Vomiting
• Headache
• Fever
• Dry skin
• Constipation
• Nausea
• Acne
• Upper respiratory tract infection

Call your child’s care team for medical advice about possible side effects.

Talk with your child’s care team about any side effects to determine the best next steps for managing them. Only your child’s care team can properly help manage side effects. You can also learn about some management tips by downloading What to Know When Starting OJEMDA Guide.

Once the treatment is stopped, your child’s hair, eyebrows, and eyelashes may grow back in their natural color.

Rash typically appeared 2 weeks after the first dose and up to 16 months after.

OJEMDA is available in 2 forms: tablets and liquid.

You and your child’s care team will determine if OJEMDA is right for your child and decide which form may be appropriate based on factors such as your child’s ability to swallow tablets.

You and your child’s care team will determine if OJEMDA is right for your child and decide which form may be appropriate based on factors such as your child’s ability to swallow tablets.

No. OJEMDA can be taken with or without food.

Yes, your child can drink or eat immediately before and after taking OJEMDA.

OJEMDA is an oral medicine that is taken once a week. It can be taken by mouth or feeding tube as prescribed by your child’s doctor. OJEMDA can be taken with or without food.

Talk to you child’s care team about using gloves when giving OJEMDA.

If your child has been prescribed OJEMDA, use it exactly as directed by your child’s care team. You should have received the Step-by-Step Guide for Preparing a Liquid Dose of OJEMDA with your child’s initial supply of OJEMDA. You can download this resource below. This guide was created to empower you with the knowledge and confidence needed to prepare and administer OJEMDA to your child. Review the instructions with your child’s care team to ensure you’re comfortable administering the medication at home.

Download the Step-by-Step Guide for Preparing a Liquid Dose of OJEMDA

Your child’s prescribed dose may require preparing more than 1 bottle of OJEMDA. If 2 bottles are required, you should always prepare and give each bottle 1 at a time. Download the Step-by-Step Guide for Preparing a Liquid Dose of OJEMDA for information on how to prepare the liquid formulation of OJEMDA.

You should never use leftover liquid OJEMDA once you have given a dose to you child. Any unused medicine must be thrown away.

If OJEMDA gets on your or your child’s skin, wash the area well with soap and water. If it gets in your or your child’s eyes, rinse the eyes well with cool water.

If your child vomits right after taking a dose of OJEMDA, give them another dose. If you are not sure if you should repeat the dose, contact your child’s care team.

Once 15 minutes have passed, the mixed medicine will thicken into a gel. It will be too thick to force through the syringe. Please do not give your child the dose if 15 minutes have passed. Call your child’s care team, your pharmacist, or EveryDay Support From Day One™ to get a replacement dose.

If a dose is missed by

• 3 days or less, the missed dose should be taken as soon as possible, and the next dose should be taken on its regularly scheduled day.
• More than 3 days, the missed dose should be skipped and the next dose should be taken on its regularly scheduled day.

Download the What to Know When Starting OJEMDA for more information on missed doses.

If your child takes too much OJEMDA, please contact your child’s care team right away.

OJEMDA may affect the way other medicines work, and other medicines may affect how OJEMDA works. Tell your child’s care team about all the medicines your child takes, including prescription and over-the-counter medicines, vitamins, and herbal supplements. They can help determine if there is a likelihood of an interaction.

Throw away the bottle, including any unused medicine and syringe, in your household trash.

Store OJEMDA at room temperature between 68 °F and 77 °F (20 °C and 25 °C) and in a dry, safe place, out of the reach of children.

Your child’s care team will determine the dosage of OJEMDA based on your child’s body surface area. Body surface area is a way to measure your child’s body to make sure they get the right amount of medicine.

Do not give your child any chipped or broken OJEMDA tablets. Do not use OJEMDA oral suspension if the safety seal under the cap is broken or missing. Call your specialty pharmacy if a replacement is needed.

Yes. Call your child’s care team, your pharmacist, or EveryDay Support From Day One™ to get a replacement dose.

It’s a program created to provide personalized support services for those who have been prescribed OJEMDA and their families. We work directly with patients and their care teams to address patients’ specific needs related to accessing OJEMDA, such as insurance coverage support, financial assistance for eligible patients, and treatment education.

EveryDay Support From Day One’s goal is to simplify the treatment journey for you and your child. We are here to help patients start and stay on OJEMDA from Day One, and every day after.

We provide

• Dedicated Patient Navigators: Your main contact who works with you and your care team to help you get treatment. We offer personalized support to address your needs
• Coverage support: We partner with your child’s doctor’s office and health insurance plan to understand your benefits and help obtain insurance coverage
• Financial assistance: We provide financial assistance programs to help eligible families pay for their child’s medicine
• Shipment and medication support: We work with our specialty pharmacy partners to ship OJEMDA directly to your home address and provide ongoing prescription and treatment support

For more information, visit EveryDaySupport.com or call 855-DAY1-BIO (855-329-1246).

Visit EveryDaySupport.com/consent to enroll online, or complete the enrollment form with your doctor in their office.

There are 2 ways to provide consent:

• Provide consent electronically at EveryDaySupport.com/consent
• Complete the Patient Authorization section (pages 3 and 4) of the enrollment form with your child’s care team

Once you and your child’s care team have completed the form, it can be printed and signed. Your child’s care provider can then submit it via fax to 855-332-9663 or secure email to info@everydaysupport.com.

Every health insurance plan is different. EveryDay Support From Day One can work with your child’s care team and health insurance plan to help you understand your benefits and help obtain coverage. To learn more or enroll

• Call a Patient Navigator at 855-DAY1-BIO/855-329-1246 from 8 AM - 8 PM ET, Monday-Friday or
• Provide consent electronically to enroll in EveryDay Support From Day One at EveryDaySupport.com/consent

The amount you pay depends on your insurance plan and type of coverage. No matter the insurance, we believe cost should not be a barrier to access. We are offering a variety of financial assistance programs to help eligible patients with coverage and affordability through EveryDay Support From Day One.

To learn more or enroll

• Call a Patient Navigator at 855-DAY1-BIO/855-329-1246 from 8 AM - 8 PM ET, Monday-Friday or
• Provide consent electronically to enroll in EveryDay Support From Day One at EveryDaySupport.com/consent

Yes, EveryDay Support From Day One offers a copay program where eligible patients with commercial prescription drug insurance coverage for OJEMDA may pay as little as $0 per month.

The first step is to verify your insurance benefits for OJEMDA. In order to do so, you can provide consent electronically to enroll in EveryDay Support From Day One at EveryDaySupport.com or you can work with your child’s care team to complete an enrollment form that can be found at EveryDaySupport.com/consent.

Yes, the program is completely free for patients who are prescribed OJEMDA. Patients only need to provide their consent to be enrolled in EveryDay Support From Day One.

Yes. Here are a few pediatric cancer advocacy foundations you can reach out to for support:

• Children’s Brain Tumor Foundation: cbtf.org
• Pediatric Brain Tumor Foundation: curethekids.org
• Momcology: momcology.org
• Team Jack Foundation: teamjackfoundation.org
• The Lilabean Foundation: lilabeanfoundation.com

These organizations are only a few of the many pediatric cancer advocacy foundations in the US.

Reach out to your child’s care team for a more comprehensive list, including local groups.