Ponatinib (Oral)

Ponatinib is a form of oral chemotherapy drug that is used to treat certain forms of chronic leukemia, where other drugs have not proved to be effective.


In the US, ponatinib is known by the brand name, Iclusig. The drug is an antineoplastic (chemotherapy) drug that is available via prescription from your treating physician. The medicine comes in tablet form.

Ponatinib is used to treat patients who are suffering from chronic Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ ALL) and chronic myeloid leukemia. This drug is used when other cancer medicines have failed to achieve good results in these patients.

Ponatinib works by disrupting the growth of the cancerous cells, allowing the body's natural defense mechanisms to destroy them. Unfortunately, normal cells can also be adversely affected by ponatinib, resulting in some side effects. It is therefore important that you discuss the pros and cons of using the drug with your doctor before your treatment commences.

Conditions treated

  • Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ ALL)
  • Chronic myeloid leukemia

Type of medicine

  • Antineoplastic
  • Tablet

Side effects

Some drugs can cause unwanted side effects, as well as being effective and beneficial in treating a particular illness. Not every patient taking ponatinib will experience all of the effects mentioned in this guide, but if you are affected, you may need to consult your doctor.

If you notice any of the side effects listed below, you must check with your GP immediately:

  • Yellow eyes or skin
  • Weakness in the arms, legs, hands, or feet
  • Unexpected weight gain or loss
  • Unusual weakness or tiredness
  • Unusual bleeding or bruising
  • Moving awkwardly or unsteadily
  • White spots, ulcers, or sores in the mouth
  • Tingling of the feet or hands
  • Sudden visual changes
  • Sudden slurring of speech
  • Sudden shortness of breath
  • Loss of coordination
  • Sore throat
  • Slow or rapid heartbeat
  • Severe vomiting, sometimes with blood
  • Severe, sudden headaches
  • Sensitivity of eyes to light
  • Dark brown or red urine
  • Black or red, tarry stools
  • Rapid, shallow breathing
  • Rapid, unexpected weight gain
  • Bleeding from cuts that won't stop
  • Pounding in the ears
  • Red, pinpoint skin spots
  • Paralysis
  • Pale skin
  • Pains in the side, stomach, or abdomen, maybe radiating to the back
  • Pain in the groin, chest, or legs, especially in the calves
  • Reddening, pain, or swelling in the legs or arms
  • Nosebleeds
  • Nervousness
  • Nausea and vomiting
  • Lower back, stomach, or side pain
  • Loss of appetite
  • Lightheadedness
  • Joint stiffness, pain, or swelling
  • Itching or skin rash
  • Irregular breathing
  • Indigestion
  • Increased vaginal bleeding or menstrual flow
  • Headache
  • Frequent urge to urinate
  • Fever
  • Rapid, pounding, or irregular heartbeat or pulse
  • Fainting
  • Extreme fatigue
  • Dry eyes
  • Dizziness
  • Dilated neck veins
  • Problems with swallowing or breathing
  • Burning, difficult, or painful urination
  • Diarrhea
  • Decreased urine output
  • Poor appetite
  • Dark urine
  • Coughing up blood
  • Cough or hoarseness
  • Constipation
  • Confusion
  • Cloudy urine
  • Clay-colored stools
  • Chills
  • Chest pain or discomfort
  • Chest pain
  • Tingling, burning, numbness, or other painful sensations
  • Blurred vision
  • Seeing blue-green halos around objects
  • Swelling or bloating of the face, lower legs, arms, hands, or feet
  • Bleeding gums
  • Bladder pain
  • Anxiety
  • Tenderness of abdomen or stomach

There are some effects that may be experienced by people taking ponatinib that do not generally require a doctor's attention, as they resolve themselves over time. Your treating specialist may also be able to suggest ways of managing or preventing these effects. However, if the following side effects continue to bother you even after a few weeks of taking the drug, you should speak to your doctor:

  • Trouble sleeping
  • Stuffy or runny nose
  • Sore throat
  • Muscle stiffness or pain
  • Muscle aches
  • Loss or lack of strength
  • Skin reactions including, itching, pain, redness, swelling, tenderness, or warmth
  • Dry skin
  • Difficulty moving
  • Constipation
  • Cracked lips
  • Back or bone pain

There may be other effects that are not included in this guide. If you notice any other odd feelings or occurrences while taking this drug, consult your GP or specialist.


You should take your prescribed ponatinib dose exactly as you are told to by your oncologist or GP. Do not take less or more of the drug or take it for longer than you have been told to. If you vary the dose or frequency, you could risk suffering side effects or compromising the efficacy of the medicine.

When you receive your prescription of ponatinib, you will be given an information leaflet. Be sure to read the contents of the leaflet carefully and ask your pharmacist or doctor if you have any queries.

You may take your tablets with food or just with water if you prefer. However, you should always take the tablets whole, without crushing, chewing or breaking them.

It is not recommended that you eat grapefruit or drink grapefruit juice while you are taking ponatinib. Some of the chemicals contained in grapefruit may affect the blood levels of ponatinib to be higher than usual, potentially raising the risk of overdose.

The dose of the drug that you are prescribed will vary between patients. You should take your medication as directed by your GP or as per the instructions on the product label. The information that follows is based on the average for this drug. If you are told to take a different dose, do not alter it unless you are told to by your treating physician.

Your dose will depend on the strength of the preparation you are given. Additionally, the number of daily doses you take, the time you leave between doses, and the duration of your course of treatment will all depend on the condition for which you are receiving treatment.

For the treatment of leukemia

  • Adults: Initially, take 45 mg daily. Your treating specialist may alter the dose if necessary.
  • Children: Your child's oncologist will determine the dose and use of ponatinib.

If you miss a dose of your medication, try to take it as soon as you can. If it is nearly time for your scheduled dose, leave out the dose you have missed and revert to your usual schedule. Do not take twice the amount you are prescribed.

Under no circumstances should you share this medication with anyone else.

Do not stop taking your prescribed dose of ponatinib unless you are told to by your doctor, even if you begin to feel much better. If you begin to feel much worse after you start taking this medicine, you should seek medical advice.


Some forms of medicine should not be taken at the same time because doing so could cause interactions to occur. However, in some cases taking two drugs together may be the best course of action in your case, and your doctor may decide to change the dose or frequency of use of one or both of your medications.

It is not recommended that ponatinib is used with any of the medicines listed below. Your doctor may decide to change your drug regimen or opt not to treat you with ponatinib:

  • Varicella virus vaccine
  • Live rubella virus vaccine
  • Live rotavirus vaccine
  • Live mumps virus vaccine
  • Live measles virus vaccine
  • Adenovirus vaccine
  • Atazanavir
  • Bacillus of calmette and guerin vaccine, live
  • Boceprevir
  • Carbamazepine
  • Ceritinib
  • Live cholera vaccine
  • Clarithromycin
  • Cobicistat
  • Conivaptan
  • Efavirenz
  • Enzalutamide
  • Fosphenytoin
  • Idelalisib
  • Indinavir
  • Live influenza vaccine
  • Itraconazole
  • Ketoconazole
  • Lopinavir
  • Nefazodone
  • Nelfinavir
  • Phenytoin
  • Live poliovirus vaccine
  • Posaconazole
  • Primidone
  • Rifabutin
  • Rifampin
  • Rifapentine
  • Ritonavir
  • Saquinavir
  • Smallpox vaccine
  • St John's Wort
  • Telaprevir
  • Telithromycin
  • Tipranavir
  • Typhoid vaccine
  • Voriconazole
  • Yellow fever vaccine

Some medication should not be used when you are eating, using tobacco, or drinking alcohol. You should discuss this aspect of your treatment with ponatinib with your doctor before you start using the drug.

In particular, grapefruit or grapefruit juice should not be consumed while you are taking this medicine.

Medical conditions

Some existing and historical medical conditions can affect how ponatinib works. Be sure to discuss your medical history in full, before you start taking ponatinib.

Note that using ponatinib with any of the drugs listed below can make these conditions worse:

  • Thrombocytopenia (low platelets in the blood)
  • Stomach or bowel problems (eg, perforation)
  • Pancreatitis
  • Neutropenia (low white blood cells)
  • Liver disease
  • Hyperuricemia (high uric acid in the blood)
  • Hypertension (high blood pressure)
  • Heart rhythm problems (eg, arrhythmia)
  • Congestive heart failure
  • Blood clotting problems
  • Bleeding problems
  • Anemia (low red blood cells)
  • Alcohol abuse

Ponatinib should be used with caution in patients with the following conditions, as to do so could make the side effects worse:

  • Stroke
  • Hyperlipidemia (high cholesterol or fats in the blood)
  • Heart or blood vessel disease
  • Heart attack
  • Diabetes

This drug should not be used in patients who are lactose intolerant. Ponatinib tablets contain lactose, which could exacerbate this illness.


When deciding to use a drug, you should take into account the benefits and risks of doing so. You should discuss this fully with your treating physician. Be sure to attend your GP clinic for regular checks while you are using ponatinib. These visits will allow your doctor to check for any unwanted effects and to make sure that the drug is working correctly.

If you have ever had a bad reaction to this or any other drug or over the counter medicine, you should mention it to your treating physician. You must also remember to mention any odd reactions that you have had to food colors, preservatives, particular food groups, or animal by-products.


Although there are no specific studies to show that ponatinib has any adverse on geriatric patients, they are more likely to suffer from side effects, including:

  • Poor appetite
  • Muscle spasm
  • Breathing problems
  • Fluid retention

Ponatinib should therefore be used with caution in elderly patients.

Pregnancy and breastfeeding

It has been shown that using this drug while you are pregnant can cause serious birth defects in the baby. You should avoid becoming pregnant while you are taking this medicine and for three months after you have completed your course of treatment. Talk to your GP about effective forms of contraception and tell your doctor immediately if you think you have fallen pregnant.

This medication can render some women infertile, meaning that they will not be able to have children following treatment. If you have concerns in this regard, be sure to discuss them with your doctor before you start taking the medicine.

Ponatinib is thought to pass into breast milk and can have harmful side effects in a nursing infant. If you are breastfeeding, you should stop doing so for the duration of your treatment with this medication and for three months afterward. During your treatment, ask your GP or midwife for advice on other feeding options for your infant. Do not express breast milk for later use.

Medical conditions

Ponatinib can cause blood clotting issues in some patients. You should mention to your doctor right away if you have any of the following symptoms:

  • Chest pains
  • Weakness in the legs, arms, or down one side of the body
  • Breathing problems
  • Trouble speaking clearly
  • Swelling or pain in the legs

If you notice any of the symptoms listed below, you should consult your GP, as these can indicate a serious liver disorder:

  • Pain in the stomach
  • Dark urine
  • Poor appetite
  • Jaundice
  • Pale stools

Ponatinib can cause heart failure and fluid retention. You must talk to your treating physician if you notice any of the following signs:

  • Rapid, unexplained weight gain
  • Chest pain and discomfort
  • Feeling unusually tired or weak
  • Breathing difficulties
  • Swelling of the feet, ankles, wrists, or hands

Ponatinib can cause pancreatitis. Check with your GP if you notice any of the signs listed below:

  • Sudden, severe sudden pain
  • Chills
  • Nausea
  • Constipation
  • Vomiting
  • Fever
  • Lightheadedness

If you experience any of the following serious side effects, you should not drive or operate machinery. Check with your doctor right away if you notice any of these effects:

  • Blurred vision
  • Problems reading
  • Pain in the eyes
  • Dryness or irritation of the eyes
  • Swelling of or around the eyes

Your doctor may refer you to an eye specialist for checks.

Some patients may experience a condition that affects the nerves (cranial or peripheral neuropathy) while taking ponatinib. Contact your doctor immediately if you notice any of these signs:

  • Increased or decreased sensitivity to touch or to pain
  • Burning, tingling, pain, or numbness in the feet, hands, legs, or feet

Ponatinib can also cause a reduction in the volume of white cells in the blood, posing a risk of infection. This medicine can also lower the number of platelets in the blood, meaning that your blood may not clot properly. If your blood count is low, you should take precautions to avoid these complications.

  • If possible, try to avoid environments where you will be exposed to people with infections. If you develop a chill or fever, cough, lower back or side pain, problematic or painful urination, you should seek medical attention.
  • Mention to your GP straight away if you notice any unexpected bruising or bleeding. Signs to look out for include tarry, black stools, blood in the stools or urine, red pinpoint spots on the skin.
  • Take extra care when brushing your teeth, flossing, or using a toothpick. Ask your dentist or dental hygienist for suggestions on other ways to clean your teeth. Be sure to mention to your dentist that you are using ponatinib if you need to attend for any routine dental treatment.
  • Keep your hands away from your nose and eyes unless your hands are newly washed to avoid the risk of transferring bacteria that could set off an infection.
  • Take extra care when using knives, razors, toenail, or fingernail cutters.
  • Do not take part in any dangerous or contact sports and avoid situations where you could sustain injury or bruising.

If you are due to undergo any form of invasive surgery or medical procedure, you must tell your treating medical team that you are taking ponatinib. It may be recommended that you stop taking the drug at least a few weeks before you have the treatment in order to avoid the risk of uncontrolled bleeding.

Ponatinib can cause blood clots and may also cause a condition of the brain called reversible posterior leukoencephalopathy syndrome. You must seek immediate medical attention if you develop any of the following signs:

  • Severe, sudden headaches
  • Fainting
  • Convulsions
  • Feeling unusually drowsy
  • Confusion
  • Visual disturbances
  • Problems with speech
  • Problems with mobility

Some patients taking ponatinib develop a serious allergic reaction called tumor lysis syndrome. Your oncologist will be able to prescribe you another drug to prevent this. If you notice any of the following signs, check with your doctor immediately:

  • Decreased urine output
  • Pain, swelling, or stiffness in the joints
  • Pain in the lower back, stomach, or side
  • Rapid weight gain
  • Swelling of the lower legs or feet
  • Feeling unusually weak or tired


Your ponatinib tablets should be stored at room temperature in a sealed container. Keep the tablets away from sources of heat, moisture, and direct sunlight. Do not refrigerate or freeze the drug.

Be sure to keep your medicine well out of the reach of children and pets. If your pet does eat any of your ponatinib tablets, you should contact your vet immediately.

Do not use any medication that has passed its use-by date or that appears damaged. Do not keep any drugs that you are no longer using. Ask your GP or pharmacist to dispose of the medicine safely for you. Do not discard tablets in your trash, and do not flush them down the drain or toilet.


Ponatinib is a prescription only antineoplastic (chemotherapy) drug. It is used to treat patients who are suffering from certain forms of chronic leukemia where other drugs have not been effective.

Although ponatinib is effective in treating leukemia, it is also a very potent drug that can cause very serious side effects in some patients. There is also a long list of medications that should not be used at the same time as this drug. For these reasons, you must be sure to discuss your medical history with your doctor, before you start using this medicine. You will be required to attend your doctor for regular progress checks during the course of your treatment with this medicine. Blood tests may be required to check for any unwanted effects that the drug may be causing.

This drug can be very harmful to the unborn baby and to nursing infants. You must tell your doctor if you are pregnant or breastfeeding before you begin taking ponatinib. Some women may become infertile after treatment with this medicine. If you are intending to start a family, you should discuss the potential implications of taking this drug with your oncologist or doctor before you begin treatment.