This is a summary of the European public assessment report (EPAR) for Venclyxto. It explains how the Agency assessed the medicine to recommend its authorisation in the EU and its conditions of use. It is not intended to provide practical advice on how to use Venclyxto.
For practical information about using Venclyxto, patients should read the package leaflet or contact their doctor or pharmacist.
- What is Venclyxto and what is it used for?
Venclyxto is a medicine for treating a blood cancer known as chronic lymphocytic leukaemia (CLL) when other treatments have failed or are unsuitable.
In patients with particular genetic changes (17p deletion or TP53 mutation) that make them unsuitable for chemo-immunotherapy, Venclyxto is used when medicines known as B‑cell receptor pathway inhibitors (ibrutinib and idelalisib) are not suitable or have failed.
In patients who do not have these genetic changes, Venclyxto is used after treatments with chemo-immunotherapy and a B‑cell receptor pathway inhibitor has failed.
Because the number of patients with CLL is low, the disease is considered ‘rare’, and Venclyxto was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 6 December 2012.
Venclyxto contains that active substance venetoclax.
- How is Venclyxto used?
Venclyxto is available as tablets to be taken by mouth once a day with a meal. The starting dose is 20 mg daily and the dose is gradually increased over 5 weeks to 400 mg. The patient should stay on treatment for a long as the patient improves or remains stable and the side effects are tolerable. If the patient experiences certain side effects, treatment may be stopped temporarily or the dose reduced.
Venclyxto should be started and supervised by a doctor with experience of cancer medicines and can only be obtained with a prescription.
- How does Venclyxto work?
The active substance in Venclyxto, venetoclax, attaches to a protein called Bcl-2. This protein is present in high amounts in CLL cancer cells, where it helps the cells survive for longer in the body and makes them resistant to cancer medicines. By attaching to Bcl-2 and blocking its actions, venetoclax causes the death of cancer cells and thereby slows the progression of the disease.
- What benefits of Venclyxto have been shown in studies?
Studies have shown that a high proportion of patients have their cancer cells partially or completely cleared following treatment with Venclyxto. In a main study of 107 previously treated patients with CLL and 17p deletion, 75% responded partially or completely to Venclyxto. In another study of 64 patients with or without 17p deletion or TP53 mutation, the response rate was 67%. Patients in this second study had all previously taken B‑cell receptor pathway inhibitors.
- What are the risks associated with Venclyxto?
The most common side effects with Venclyxto (seen in more than 1 in 5 people) are reduced neutrophils (a type of white blood cell), diarrhoea, nausea, anaemia (low red blood cell counts), nose and throat infection, tiredness, high levels of phosphate in the blood, vomiting and constipation.
The most common serious side effects (seen in more than 2 in 100 people) are pneumonia (lung infection), fever associated with reduced neutrophils and tumour lysis syndrome (a complication caused by breakdown of cancer cells). For the full list of side effects reported with Venclyxto, see the package leaflet.
Venclyxto must not be used with medicines known as ‘strong CYP3A inhibitors’ during the early stages of treatment and must also not be used with St. John’s wort (a herbal preparation used to treat anxiety and depression).
- Why is Venclyxto approved?
A high proportion of patients respond to Venclyxto after other treatments have failed or are unsuitable.Studies showed patients with particular genetic mutations (17p deletion or TP53 mutations) that make them unsuitable for chemo-immunotherapy responding well to treatment. In addition, a high response rate was seen in patients whose previous treatment with ibrutinib or idelalisib failed.
Regarding safety, the medicine’s side effects are considered acceptable. Although there is a risk of tumour lysis syndrome, a complication that occurs when the cancer cells are being destroyed too quickly, this risk can be contained through preventive measures, such as increasing the dose gradually or reducing the dose, if needed.
The Agency’s Committee for Medicinal Products for Human Use (CHMP) noted that although a small number of patients have been studied so far Venclyxto's benefits outweigh its risks and recommended its approval in the EU.
Venclyxto has been given ‘conditional approval’. This means that there is more evidence to come about the medicine. Every year, the European Medicines Agency will review any new information that becomes available and this summary will be updated as necessary.
- What information is still awaited for Venclyxto?
As Venclyxto has been granted a conditional approval, the company that markets the medicine will provide further data on its benefits and risks from an ongoing study of patients whose previous treatment with ibrutinib or idelalisib had failed.
- What measures are being taken to ensure the safe and effective use of Venclyxto?
The company that markets Venclyxto will provide more data on the medicine’s overall safety. Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Venclyxto have also been included in the summary of product characteristics and the package leaflet.
- Other information about Venclyxto
The European Commission granted a conditional marketing authorisation valid throughout the European Union for Venclyxto on 5 December 2016.
For more information about treatment with Venclyxto, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.
The summary of the opinion of the Committee for Orphan Medicinal Products for Venclyxto can be found on the Agency’s website.
This EPAR was last updated on 21/12/2016 .
05/12/2016 Venclyxto -EMEA/H/C/004106 --
- Annex I - Summary of product characteristics
- Annex IIA - Manufacturing-authorisation holder responsible for batch release
- Annex IIB - Conditions of the marketing authorisation
- Annex IIIA - Labelling
- Annex IIIB - Package leaflet
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Venclyxto monotherapy is indicated for the treatment of chronic lymphocytic leukaemia (CLL) in the presence of 17p deletion or TP53 mutation in adult patients who are unsuitable for or have failed a B cell receptor pathway inhibitor.
Venclyxto monotherapy is indicated for the treatment of CLL in the absence of 17p deletion or TP53 mutation in adult patients who have failed both chemoimmunotherapy and a B cell receptor pathway inhibitor.
Changes since initial authorisation of medicine
Initial marketing-authorisation documents
This medicine is approved for use in the European Union