Myelofibrosis (MF) is a rare form of cancer that affects thousands of people and can affect how your body makes blood cells. MF will usually worsen over time and the severity of symptoms can vary from person to person. People with MF can also have anemia (low red blood cells) and may need blood transfusions.
Ojjaara (oh-JAR-uh) was approved in 2023 as the first medicine to treat people with MF and anemia.
How Does Ojjaara Work?
Myelofibrosis (MF) starts in your bone marrow, which is responsible for making blood cells. It happens when there is a problem with one of your genes, and it causes scarring in your bone marrow. MF can occur by itself or if you have another type of blood/bone marrow cancer such as leukemia. The disease can also affect specific organs including the spleen and can cause physical symptoms as well. Common symptoms of MF are:
- Tiredness (due to low red blood cells)
- Infections (due to low white blood cells)
- Bruising/bleeding (due to low platelet levels)
- Swollen liver or spleen
- Night sweats
- Weight loss
- Itching
- Stomach pain
- Bone pain
Ojjaara is a Janus kinase (JAK) inhibitor. JAKs are proteins inside certain cells, including cells of the bone marrow. In MF, the JAKs become overactive and cause scar tissue to form in the bone marrow, which can lead to less blood cells being formed. Ojjaara stops the overactive JAKs and also stops a protein called activin A receptor type 1 (ACVR1) in your liver cells, which increases the number of red blood cells in your body.
How Do I Take It?
Ojjaara is taken by mouth once a day, with or without food. Do not cut, crush, or chew the tablet. Ojjaara comes in three doses: 100 milligrams, 150 milligrams, and 200 milligrams. The recommended dose is 200 milligrams per day. If you have liver problems, your health care provider may start you on a lower dose of Ojjaara.
How Was Ojjaara Studied?
Two studies were conducted for Ojjaara. The first study looked at people who have taken JAK inhibitors in the past who were experiencing MF symptoms and had anemia. People received either Ojjaara or danazol, which is a medicine used to treat people with MF. The study looked at the percentage of people who had a 50% or greater reduction in their Myelofibrosis Symptom Assessment Form (MFSAF) score from baseline to week 24. The MFSAF looked at people’s symptoms and quality of life. A higher MFSAF meant that a person had more MF symptoms. The median age was 71, and 63% of people in the study were men.
The second study looked at people who have never taken a JAK inhibitor. People received either Ojjaara or ruxolitinib, another JAK inhibitor, for 24 weeks. The study looked at the percentage of people who had 35% or greater reduction in their spleen volume. The average age of people in the study was 65, and 57% of people in the study were men.
What Benefits Were Seen?
The results of the first study showed that Ojjaara improved MF symptoms including anemia and spleen size. People taking Ojjaara were more likely to experience a 50% or greater MFSAF reduction compared to people taking danazol (25% vs. 9%).
The results of the second study showed that Ojjaara (26.5%) showed a similar reduction in spleen volume compared to ruxolitinib (29%).
How Long Does It Take to Work?
In clinical studies, Ojjaara’s benefits were seen after a few months of taking the medicine. Your health care provider will monitor you while you are taking the medicine and may adjust your dose or switch you to another medicine depending on how you tolerate Ojjaara.
What Are the Side Effects and How Can I Manage Them?
The most common side effects seen with Ojjaara are listed below.
- Low platelet count in the blood
- Bleeding
- Bacterial infections
- Tiredness
- Dizziness
- Diarrhea
- Nausea
Your health care provider will monitor you while taking Ojjaara, including monitoring your blood tests.
Call your health care provider if you experience bleeding while taking Ojjaara. This can include coughing up blood; blood in your vomit; red, pink, or brown urine; or red/black stools.
Call your health care provider if you start to feel ill or have symptoms that are not usual to you. You may be at a higher risk of developing bacterial infections while taking Ojjaara.
Ojjaara may cause you to feel tired or dizzy. Do not drive or operate heavy machinery until you know how Ojjaara affects you.
Diarrhea may occur while you are taking Ojjaara. To limit diarrhea, you can try simple steps such as eating bland foods like rice, bananas, and toast, drinking plenty of water, and lowering the amount of caffeine you are drinking. Call your health care provider if your diarrhea symptoms are not improving or are getting worse.
If you experience nausea, make sure you are drinking plenty of water to prevent yourself from becoming dehydrated, and call your health care provider if your symptoms are not getting better. You can also take these steps to lessen the symptoms:
- Avoid greasy food
- Eat bland food such as toast and crackers
- Avoid strong smells
- Eat slowly
What Type of Drug Interactions Can Happen?
Taking Ojjaara with medicine called rifampin, which is used to treat tuberculosis, can cause the blood levels of Ojjaara to increase and can increase the risk of side effects.
Taking Ojjaara with a medicine called rosuvastatin (Crestor), which is used for high cholesterol, can increase blood levels of rosuvastatin.
These may not be the only medicines that interact with Ojjaara. There are certain proteins that metabolize the medicines you take. Taking certain medicines with Ojjaara can affect the blood levels of Ojjaara and vice versa. Talk to your health care provider about all the prescription and over-the-counter medicines, vitamins, and supplements you take.
Is There a Cost-Savings Program?
There is a copay assistance program and patient assistant program that may allow you to pay as little as $0 for your Ojjaara prescription. Whether you are eligible depends on the type of insurance you have. You can find out more at ojjaara.com/savings-and-support/reimbursement-support or by calling 844-447-5662.
