Iclusig
Authorised
This medicine is authorised for use in the European Union
ponatinib
MedicineHumanAuthorised
- Application under evaluation
- CHMP opinion
- European Commission decision
Overview
Iclusig is a cancer medicine used on its own to treat the following types of leukaemia (cancer of white blood cells):
- chronic myeloid leukaemia (CML) in adults at any stage of the disease (chronic, accelerated or blast phase), and in children from six years of age with chronic phase CML (CP-CML). It is used when treatment with dasatinib or nilotinib (other cancer medicines) has not worked, or when these medicines cannot be used because of side effects and imatinib (another cancer medicine) is not suitable, or when the cancer has a change in the BCR-ABL1 gene called T315I mutation;
- acute lymphoblastic leukaemia (ALL) in adults when the cancer is Philadelphia chromosome-positive (Ph+). Ph+ means that some of the patient’s genes have rearranged to form an abnormal chromosome that leads to the development of leukaemia. Iclusig is used when treatment with dasatinib has not worked, or when dasatinib cannot be used because of side effects and imatinib is not suitable, or when the cancer has a change in the BCR-ALB1 gene called T3151 mutation;
Iclusig is used together with reduced-intensity chemotherapy, which is less toxic than standard chemotherapy, to treat adults with newly diagnosed ALL when the cancer is Ph+.
Iclusig contains the active substance ponatinib.
Iclusig can only be obtained with a prescription, and treatment should be started by a doctor who is experienced in the diagnosis and treatment of leukaemia.
Iclusig is available as tablets and capsules to be taken by mouth once a day. Treatment is continued for as long as the patient benefits from it. If a patient develops certain severe side effects, the doctor may decide to reduce the dose or stop treatment temporarily or permanently. Patients with newly diagnosed Ph+ ALL first receive Iclusig together with reduced-intensity chemoterapy, and then Iclusig on its own.
Because Iclusig can cause blood clots or blockages in arteries and veins, the doctor should assess the patient’s heart and blood vessels health before starting and during treatment. Treatment should be stopped immediately if a blockage develops in an artery or vein.
For more information about using Iclusig, see the package leaflet or contact your doctor or pharmacist.
The active substance in Iclusig, ponatinib, belongs to a group of medicines called ‘tyrosine kinase inhibitors’. These compounds act by blocking enzymes known as tyrosine kinases. Ponatinib acts by blocking a tyrosine kinase called BCR-ABL. This enzyme is found on the surface of leukaemia cells where it is involved in stimulating the cells to divide uncontrollably. By blocking BCR‑ABL, Iclusig helps to control the growth and spread of leukaemia cells.
Iclusig has been investigated in one main study involving 449 adults with CML or Ph+ ALL and who were intolerant or resistant to treatment with dasatinib or nilotinib, or had the T315I mutation. In the study, Iclusig was not compared with another treatment. The response to treatment was assessed by measuring the proportion of patients who had a major haematological response (when the number of white blood cells returns to normal or there is no evidence of leukaemia) or a major cytogenetic response (when the proportion of white blood cells containing the Philadelphia chromosome falls to below 35%).
The study showed that treatment with Iclusig led to clinically relevant responses in all groups of patients. Among patients with CML in the chronic phase, around 54% (144 out of 267) had a major cytogenetic response. In the accelerated phase, around 58% (48 out of 83) had a major haematological response, while in the blast phase, around 31% (19 out of 62) had a major haematological response. Among patients with Ph+ ALL, around 41% (13 out of 32) had a major haematological response.
Data from an ongoing study involving 10 children between six and 18 years of age with CP-CML who were intolerant or resistant to treatment with dasatinib, imatinib or nilotinib, or had the T315I mutation has shown that, within 12 months of treatment with Iclusig, 90% (9 out of 10) of patients had a major cytogenetic response.
A main study involving 245 adults showed that Iclusig in combination with reduced-intensity chemotherapy is effective in treating Ph+ ALL. In this study, participants received reduced-intensity chemotherapy with either Iclusig or imatinib.
The main measure of effectiveness was the proportion of participants who had a complete response (no detectable signs of cancer) and no minimal residual disease (when a very small number of cancer cells remain in the body after treatment). At the end of induction treatment with Iclusig and reduced-intensity chemotherapy, about 34% of participants (53 out of 154) had a complete response with no minimal residual disease, compared with about 17% of participants (13 out of 78) treated with imatinib and reduced-intensity chemotherapy. Information on how long patients lived overall was not yet available.
Studies carried out with Iclusig are described in more detail in the published assessment reports.
For the full list of side effects and restrictions with Iclusig, see the package leaflet.
In the main study involving 449 patients adults with CML or Ph+ ALL, the most common serious side effects with Iclusig (which may affect more than 2 in 100 people) include pneumonia (infection of the lungs), pancreatitis (inflammation of the pancreas), pyrexia (fever), abdominal (belly) pain, myocardial infarction (heart attack), atrial fibrillation (irregular and uncoordinated contractions of the upper chambers of the heart), peripheral arterial occlusive disease (problem with blood flow in the arteries), anaemia (low levels of red blood cells), angina pectoris (pains to the chest, jaw and back due to problems with blood flow to the heart), decreased blood levels of platelets (components that help the blood to clot), febrile neutropenia (fever with a low level of neutrophils, a type of white blood cell), hypertension (high blood pressure), coronary artery disease (heart disease caused by narrowing or blockage of blood vessels supplying the heart muscle), congestive cardiac failure (when the heart does not work as well as it should), cerebrovascular accident (stroke), sepsis (when bacteria and their toxins circulate in the blood, leading to organ damage), cellulitis (inflammation of the deep skin tissue), acute kidney injury (kidney damage), urinary tract infection (infection of the parts of the body that collect and pass out urine) and increased levels of lipase (an enzyme).
Arterial occlusions (clots or blockages in the arteries) may affect more than 1 in 5 people, with serious arterial occlusions affecting 1 in 5 people. Serious venous occlusions (clots or blockages in the veins) may affect up to 1 in 20 people. Venous thromboembolic reactions (problems due to blood clots in the veins) may affect up to 1 in 10 people.
The European Medicines Agency decided that Iclusig’s benefits are greater than its risks and it can be authorised for use in the EU. Iclusig was shown to be an effective treatment for adults with CML, children from six years of age with CP-CML and in adults with Ph+ ALL, who have limited treatment options.
Regarding safety, the side effects with Iclusig were broadly similar to those of other tyrosine kinase inhibitors and mostly manageable with dose reduction or dose delay. There is a risk of problems resulting from blood clots or blockages in arteries or veins with Iclusig, including heart attacks and strokes. This risk can be reduced by identifying and treating conditions that may contribute to the risk, both before and during treatment. These conditions include high blood pressure and raised cholesterol.
The company that markets Iclusig must submit the final results of the study in adults with newly diagnosed Ph+ ALL to confirm its safety and effectiveness.
Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Iclusig have also been included in the summary of product characteristics and the package leaflet.
As for all medicines, data on the use of Iclusig are continuously monitored. Side effects reported with Iclusig are carefully evaluated and any necessary action taken to protect patients.
Iclusig received a marketing authorisation valid throughout the EU on 1 July 2013.
Product information
Latest procedure affecting product information:X/0000296489
15/07/2026
This medicine’s product information is available in all official EU languages.
Select 'available languages' to access the language you need.
Product information documents contain:
- summary of product characteristics (annex I);
- manufacturing authorisation holder responsible for batch release (annex IIA);
- conditions of the marketing authorisation (annex IIB);
- labelling (annex IIIA);
- package leaflet (annex IIIB).
Product details
- Name of medicine
- Iclusig
- Active substance
- ponatinib
- International non-proprietary name (INN) or common name
- ponatinib
- Therapeutic area (MeSH)
- Leukemia, Myeloid
- Leukemia, Lymphoid
- Anatomical therapeutic chemical (ATC) code
- L01EA05
Pharmacotherapeutic group
- Antineoplastic agents
- Protein kinase inhibitors
Therapeutic indication
Iclusig is indicated as monotherapy in adult patients with
- chronic phase, accelerated phase, or blast phase chronic myeloid leukaemia (CML) who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation
- Philadelphia chromosome positive acute lymphoblastic leukaemia (Ph+ ALL) who are resistant to dasatinib; who are intolerant to dasatinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation.
Iclusig is indicated in combination with reduced-intensity chemotherapy in adult patients with newly diagnosed Ph+ ALL (see section 5.1).
Iclusig is indicated as monotherapy in paediatric patients 6 years of age or older with
- chronic phase chronic myeloid leukaemia (CP-CML) who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation.
See sections 4.2 for the assessment of cardiovascular status prior to start of therapy and 4.4 for situations where an alternative treatment may be considered.
News on Iclusig
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