What is Venclyxto - Venetoclax and what is it used for?
Venclyxto is a medicine to treat a blood cancer known as chronic lymphocytic leukemia (CLL) when other treatments have failed or are unsuitable.
In patients with particular genetic alterations (deletion 17p or TP53 mutation) that make them not suitable for chemoimmunotherapy, Venclyxto is used when medicines known as B-cell receptor inhibitors (ibrutinib and idelalisib) are not suitable or have failed.
In patients who do not have such genetic alterations, Venclyxto is used after chemoimmunotherapy treatments and a B-cell receptor pathway inhibitor have 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 the active substance venetoclax.
How is Venclyxto - Venetoclax used?
Venclyxto is available as tablets taken by mouth once a day during a meal. The starting dose is 20 mg a day and is gradually increased to 400 mg over 5 weeks. The patient must remain on treatment until he improves or remains stable and the side effects are tolerable. If the patient feels some side effects, you can temporarily stop the treatment or reduce the dose.
Venclyxto must be started and supervised by a doctor experienced in the use of anticancer medicines and can only be obtained with a prescription.
How does Venclyxto - Venetoclax work?
The active substance in Venclyxto, venetoclax, binds to a protein called Bcl-2. This protein is present in high quantities in CLL tumor cells and helps them survive longer in the body, making them resistant to anticancer medicines. By binding to Bcl-2 and blocking its action, venetoclax causes the death of cancer cells, thus slowing the progression of the disease.
What benefit has Venclyxto - Venetoclax shown during the studies?
Studies have shown that in a high percentage of patients, tumor cells are partially or completely eliminated following treatment with Venclyxto. In one main study involving 107 previously treated CLL and 17p deletion patients, 75% responded partially or completely to Venclyxto. In another study of 64 patients with or without a 17p deletion or TP53 mutation, the response rate was 67%. The patients of this second study had all previously taken inhibitors of the B cell receptor pathway.
What are the risks associated with Venclyxto - Venetoclax?
The most common side effects of Venclyxto (which may affect more than 1 in 5 people) are reduction of neutrophils (a type of white blood cell), diarrhea, nausea, anemia (low red blood cell count), infection of the nose or throat, tiredness, high levels of phosphate in the blood, vomiting and constipation.
The most common serious side effects (which may affect more than 2 in 100 people) are pneumonia (lung infection), fever associated with decreased neutrophils and tumor lysis syndrome (a complication caused by the degradation of cancer cells). For the full list of all side effects reported with Venclyxto, see the package leaflet.
Venclyxto should not be used with medicines known as "potent CYP3A inhibitors" during the early stages of treatment and should not be used with St. John's wort (a plant preparation used to treat anxiety and depression).
Why has Venclyxto - Venetoclax been approved?
A high percentage of patients respond to Venclyxto after other treatments have failed or proved to be unsuitable. Studies have shown that patients with particular genetic mutations (deletion 17p or TP53 mutations), which make them unsuitable for chemoimmunotherapy, respond well to treatment. Furthermore, a high response rate was observed in patients in whom previous treatment with ibrutinib or idelalisib had failed.
Regarding safety, the side effects of the medicine are considered acceptable. Although there is a risk of tumor lysis syndrome, a complication that occurs when cancer cells are destroyed too quickly, this risk can be contained by preventive measures, such as a gradual increase in dose or reduction, if necessary.
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 are greater than its risks and recommended that it be approved for use in the EU .
Venclyxto has obtained a "conditional approval". This means that additional data on the medicine should be provided. Every year the European Medicines Agency will review the new information available and this summary will be updated accordingly.
What information is still awaited for Venclyxto?
Since a conditional approval was issued for Venclyxto, the company that markets the medicine will provide additional data on its benefits and risks from an ongoing study in patients whose previous treatment with ibrutinib or idelalisib had failed.
What measures are being taken to ensure the safe and effective use of Venclyxto - Venetoclax?
The company that markets Venclyxto will provide more data on the overall safety of the medicine. 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.
More information on Venclyxto - Venetoclax
The full EPAR for Venclyxto can be found on the Agency's website: ema.europa.eu/Find medicine / Human medicines / European public assessment reports. For more information about Venclyxto therapy, 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 is available on the Agency's website: ema.europa.eu/Find medicine / Human medicines / Rare disease designation.
