Komzifti (ziftomenib) is a once-daily oral medicine that was approved by the FDA in November 2025 to treat a type of blood cancer called acute myeloid leukemia (AML) in adults. People with AML usually receive chemotherapy, radiation therapy, stem cell transplant, and targeted medicines to keep the cancer from coming back. Komzifti is an option for people with AML that has come back (relapsed) or not responded (refractory) to previous therapy and who have a certain type of abnormal gene called a nucleophosmin 1 (NPM1) mutation.
How Does Komzifti Work in AML?
Komzifti is a type of medicine called a targeted therapy. It blocks a specific protein called menin. People with AML who have an NPM1 mutation can have abnormal activity of menin and KMT2A, another protein associated with menin. The interaction between menin and KMT2A can cause normal blood cells to become cancer cells. Komzifti blocks the protein interaction of menin and KMT2A so that normal blood cells can develop.
How Do I Take Komzifti?
Komzifti is a capsule that is taken by mouth once daily around the same time each day. Komzifti should be taken on an empty stomach, at least one hour before or two hours after a meal. Do not open, break, or chew the capsules. Swallow them whole.
Your health care provider may choose to lower your dose, pause, or stop Komzifti completely if you have side effects. Tell your health care provider about any side effects you have.
Komzifti can interact with certain medicines. Your dose of Komzifti may need to be lowered if you are taking certain medicines with it, or you may need to avoid taking certain medicines while you are on Komzifti. Tell your health care provider about all the medicines or supplements that you are taking.
How Was Komzifti Studied?
A clinical study called KOMET-001 was done to look at the benefits and side effects of Komzifti in people with relapsed or refractory AML with NPM1 mutations. The people in the study had to have either tried other therapies in the past or not be able to use other treatments for various reasons.
The main goal of the study was to see how many people with AML went into complete remission with full or partial blood count recovery after treatment with Komzifti. Complete remission is when there are no signs of cancer and blood counts return to normal. Complete remission with partial blood count recovery is when there are no signs of cancer, but some blood counts do not return to normal. The study also looked at changes in the need for red blood cell and platelet transfusions.
The study included a total of 112 people. Everyone took three capsules (600 mg) of Komzifti by mouth once daily. Below are details about the people in the study:
- Ages ranged from 22 to 86 years, with the median (middle) age being 69 years.
- 63% were 65 years or older, and the rest were younger than 65.
- 56% of the people were women, and the other 44% were men.
- Most were White (79%), 4% were Asian, 2% were Black or African American, and 16% were other or unknown race.
- Previous treatments ranged from one to seven, with the median number (middle when arranged from lowest to highest) being two.
- 26 people (23%) had a prior stem cell transplant.
People took Komzifti for anywhere from 0.1 to 41.2 months, with the median (middle) being 4.2 months. People took Komzifti until one of the following happened:
- AML got worse.
- They got serious side effects.
- They decided to stop.
- They passed away.
- The study ended.
What Benefits Were Seen With Komzifti?
Komzifti improved AML in some people. Below is a summary of the benefits seen with Komzifti:
- 24 people (21.4%) achieved either a complete remission or a complete remission with partial blood count recovery.
- Remissions lasted around five months.
- Of those who achieved a complete remission or a complete remission with partial blood count recovery, 21 people (88%) achieved it within six months of starting treatment.
- Of the 66 people who needed red blood cell or platelet transfusions at the start of the study, 14 (21%) no longer needed transfusions during any 56-day period after the start of the study.
Keep in mind that your results may differ from what was seen in clinical studies.
How Long Does It Take for Komzifti to Work?
Among the people in the study who had a complete remission or a complete remission with partial blood count recovery, the time to achieve this ranged from 0.9 to 15 months, with the middle value being 2.7 months.
As long as you show signs of improvement without serious side effects, your health care provider will probably continue Komzifti for at least six months to allow time for it to work.
How Can I Prevent and Manage Side Effects?
Side effects are common with medicines that treat cancer, including Komzifti. It is important to tell your health care provider about any side effects you have during your Komzifti treatment or after it is done. They can help determine if your side effects are serious or guide you on how to manage the side effects. Some common side effects of Komzifti include:
- Infections
- Bleeding
- Diarrhea or nausea
- Tiredness
- Arm and leg swelling
- Muscle, bone, or joint pain
- Itching
- Fever with low white blood cells
- High liver enzymes
Your health care provider will likely perform blood tests to check certain blood cell counts and electrolyte levels (potassium and magnesium) before you start and while you are taking Komzifti. They will also monitor your heart rhythm to check for problems while you take Komzifti. Make sure to keep all your appointments with your health care provider so they can monitor your improvements or side effects.
To help limit nausea, you can try simple methods such as eating small meals or eating bland foods such as toast or crackers. Your health care provider may also prescribe medicine to help manage nausea and vomiting.
Komzifti may cause low levels of white blood cells (neutropenia). This can increase your risk of infection. Be sure to minimize your risk of infection by washing your hands and avoiding contact with anyone who is sick. Contact your health care provider right away if you have signs or symptoms of an infection, such as fever or chills.
Komzifti may also cause low levels of platelets (thrombocytopenia). Sometimes, this can increase your risk for bleeding. Contact your health care provider right away if you have unexpected bleeding or bruising.
Komzifti may cause a serious side effect called differentiation syndrome. This happens when blood cells mature too quickly, causing inflammation and fluid buildup. Differentiation syndrome can lead to death if not treated. It can happen as early as three days after starting Komzifti or later during treatment. Your health care provider will monitor you closely to check for this. Tell your health care provider right away or get emergency help if you have a fever, joint or bone pain, dizziness, shortness of breath, trouble breathing, cough, chest pain, fast weight gain, rash, reduced need to pee, or swelling of your hands, feet, ankles, or legs. Your health care provider will likely start you on a type of medicine called a steroid if you get differentiation syndrome.
These are not all the possible side effects of Komzifti. Contact your health care provider for medical advice about side effects if you are having symptoms that bother you. You can also report side effects to the FDA at 800-FDA-1088 (800-332-1088). If you have certain side effects while you use Komzifti, your health care provider may need to pause your doses or even stop your medicine altogether. This will depend on the side effect you have, how severe it is, and what treatment is needed. If your Komzifti needs to be paused, it is possible that your health care provider may restart the medicine once your side effects are treated.
What Are Other Unique Considerations to Be Aware Of?
You should talk with your health care provider about any medical conditions you have had in the past, particularly if you have had any heart problems. Komzifti can cause a dangerous heart condition called QT prolongation. If you have had certain heart problems in the past, such as QT prolongation, heart failure, or issues with your electrolyte balance, Komzifti may not be safe to take. Your health care provider will review your health history with you to make sure Komzifti is safe for you to take. You will have a test called an electrocardiogram (EKG) and blood tests to measure your potassium and magnesium levels before and during your treatment. Tell your health care provider right away if you feel faint, dizzy, have shortness of breath, or have a fast or abnormal heartbeat.
Komzifti can harm a fetus. If you are able to get pregnant, confirm that you are not pregnant before starting Komzifti. Talk to your health care provider about effective birth control options. They should be used while you take Komzifti and for at least six months after your last dose. If you can get your partner pregnant, use effective birth control to prevent pregnancy during treatment with Komzifti and for six months after the last dose. Talk to your health care provider if you have questions or concerns about this.
What Kind of Drug Interactions May Happen?
Many other medicines can interact with Komzifti. Do not start any new medicine until you check with your health care provider.
Komzifti should not be used with other medicines that can cause QT prolongation or affect your heart rhythm.
Some medicines can increase or decrease the blood levels of Komzifti, which could increase your risk for side effects or make Komzifti not work as well. These medicines are called CYP3A4 inhibitors and inducers.
Medicines that lower stomach acid can lower blood levels of Komzifti, which could make it not work as well.
- You should not take medicines called proton pump inhibitors (PPIs), such as omeprazole (Prilosec), with Komzifti.
- If you take a medicine called an H2 receptor blocker, such as famotidine (Pepcid), you should take Komzifti either two hours before or 10 hours after the H2 receptor blocker.
- If you take antacids such as calcium carbonate (Tums), take Komzifti either two hours before or two hours after the antacid.
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, could cause QT prolongation, or interact with Komzifti.
Is There Any Cost Assistance Available?
There is a patient support program offered by the drugmaker called Kura RxKonnect that may help you afford Komzifti. Your health care provider can help you get started. You can find out more at this website or by calling 844-587-2777.
