Ojemda (tovorafenib) is a once-weekly oral medicine that was approved by the FDA in April 2024 for the treatment of a brain cancer known as pediatric low-grade glioma (LGG). People with LGG usually receive surgery to remove the tumor. Chemotherapy, radiation therapy, and targeted medicines are used to keep the cancer from coming back and to shrink the tumor. Ojemda is a treatment option for people with pediatric LGG that has come back (relapsed) or not responded (refractory) to previous therapy and who have a certain type of abnormal BRAF gene.
How Does Ojemda Work for Low-Grade Glioma?
Ojemda is a targeted therapy known as a type 2 RAF kinase inhibitor. It works by blocking certain proteins, called RAF kinases, that send growth signals to cancer cells. It works in people with LGG who have a genetic change in the BRAF gene called a BRAF fusion or a BRAF V600E mutation. When these kinases are blocked, it may help shrink the tumor and stop cancer cells from growing and multiplying.
How Do I Take Ojemda?
Ojemda is an oral medicine that is taken by mouth one time each week on the same day of the week. Ojemda can be taken with or without food.
Ojemda comes as a tablet that is swallowed whole. Do not cut, crush, or chew the tablets. If you or your child has difficulty swallowing tablets, Ojemda also comes as an oral suspension. An oral dosing syringe comes with the medicine and should be used to give the mixture by mouth. The mixture should be given within 15 minutes of preparing it. If not used within 15 minutes, throw it away.
Read the “Instructions for Use” that comes with Ojemda the first time you take this medicine and each time you get a refill.
How Was Ojemda Studied for Low-Grade Glioma?
A clinical study called FIREFLY-1 was done to see if Ojemda was safe and effective for treating relapsed or refractory pediatric LGG. Ojemda was not compared to any other medicine or placebo. People taking Ojemda knew that they were receiving Ojemda.
The study included a total of 76 people aged 2-21 years with a diagnosis of LGG with a BRAF mutation that was progressive (getting worse) and had at least one measurable tumor. Those whose tumors had other abnormal gene mutations or who had a condition called neurofibromatosis type 1 (NF1) were excluded from the study. People tried an average of three other therapies (range, 1 to 9) before starting the study. The most common tumor locations were in the optic pathway (a pathway that carries information from your eyes to your brain so that you can see; 51%) and deep midline structures (important areas in the middle of the brain; 12%).
The average age of people in the study was 9 years. Over half of the people were male (53%), and 47% were female. Most people were White (61%), 7% were Asian, 2.6% were Black or African American, 3.9% were of multiple races, and 26% were of another race or their race was not reported. Some (3.9%) were of Hispanic or Latino ethnicity.
Everyone in the study took Ojemda once weekly and continued until the cancer got worse or they had a side effect that was not manageable. Every three months, people had an MRI scan done to check the status of their cancer. An independent group of experts reviewed the scan results to see how well the treatment worked, based on specific criteria
The study measured the treatment’s effectiveness using the overall response rate. This means the percentage of patients whose tumors completely disappeared on scans (complete response), shrank significantly (partial response), or shrank by a smaller amount (minor response).
What Benefits Were Seen With Ojemda?
Ojemda resulted in an overall response rate (number of people who had a response to Ojemda) of 53%. Twenty-nine people had a partial response, and 11 people had a minor response.
Your results may differ from what was seen in clinical studies.
How Long Does It Take for Ojemda to Work?
The time to response for most people ranged from 1.6 to 17.5 months, with the middle value being 5.4 months.
How Do I Know if Ojemda Is Working?
Your health care provider will regularly check how well Ojemda is working for your cancer by looking at the results of tests such as blood tests and MRI scans. Take Ojemda exactly as your health care provider tells you to. Do not change your dose or stop taking Ojemda unless your health care provider tells you to.
How Can I Prevent and Manage Side Effects?
Side effects are common with medicines that treat cancer, including Ojemda. It is important to tell your health care provider about any side effects you have during or after taking Ojemda. Some common side effects of Ojemda include:
- Rash, hair color changes, dry skin, acne
- Infections
- Nausea or vomiting
- Tiredness
- Headache
- Low white blood cells, low red blood cells, and low platelets
- High liver enzymes
Your health care provider will perform blood tests to check for any abnormalities while you take Ojemda. Make sure to keep all your appointments with your health care provider to get your blood checked. If abnormal blood tests occur, your health care provider may decide to interrupt your treatment until they return to normal levels.
If you have a headache, using an over-the-counter (OTC) pain reliever may help. Talk with your health care provider about which OTC product may be best for you.
To help limit nausea, you can try simple methods such as eating bland foods like toast or crackers. Your health care provider may also prescribe medicine to help manage nausea and vomiting.
Ojemda may cause your skin to be sensitive to sunlight, sunlamps, or tanning beds. You may get a severe sunburn from exposure to this light. To protect against sunburn, you should limit the time you spend in the sun and wear sunscreen, a hat, and clothing that covers your skin while you are taking Ojemda.
Liver damage, also called hepatotoxicity, can happen when taking Ojemda. Your health care provider will do blood tests to monitor how well your liver is working before and during treatment. Contact your health care provider if you notice any signs of liver problems:
- Yellowing of your skin or the whites of your eyes (also called jaundice)
- Dark-colored urine (pee)
- Nausea or vomiting
- Loss of appetite
- Feeling very tired
- Bruising
- Bleeding
- Stomach or belly pain
Ojemda may cause low levels of white blood cells (neutropenia), which can increase your risk of infection. Contact your health care provider right away if you have signs or symptoms of an infection, such as fever or chills. Ojemda may also cause low levels of platelets (thrombocytopenia). Contact your health care provider right away if you have unexpected bleeding or bruising. Your health care provider will do blood tests to monitor your blood cell counts before and during treatment with Ojemda.
If your side effects get worse or do not let up, talk with your health care provider.
What Kind of Drug Interactions May Happen?
Many other medicines can interact with Ojemda. Do not start any new medicine until you check with your health care provider.
Some medicines can increase or decrease the blood levels of Ojemda, which could increase your risk for side effects or make Ojemda not work as well. These medicines are called CYP2C8 inducers and inhibitors.
Ojemda can raise or lower the blood levels of other medicines, which could increase your risk for side effects or make other medicines not work as well. These medicines are called CYP3A4 inducers and inhibitors. For example, if you are taking Ojemda, birth control methods that contain hormones may not work as well, which may lead to pregnancy. You should use nonhormonal birth control methods during treatment with Ojemda and for 28 days after your last dose of Ojemda.
Tell your health care provider about all the prescription and OTC medicines and supplements you are taking. Your health care provider can tell you if you are taking a medicine or supplement that could affect CYP3A4 or CYP2C8 or interact with Ojemda.
Is There Any Cost Assistance Available for Ojemda?
There is a patient support program offered by the drugmaker that may help you afford Ojemda. Your health care provider can help you get started. You can find out more at www.ojemda.com/support-from-day-one or by calling 855-329-1246.