Venetoclax (EU: Venclyxto, U.S.: Venclexta) for the treatment of chronic lymphocytic leukemia (CLL)

Introduction

Venetoclax (trade name EU: Venclyxto, U.S.: Venclexta) has been approved in Germany since December 2016 for use on its own in the treatment of chronic lymphocytic leukemia (CLL) in adults. Since October 2018 it has been an option for patients who have already had treatment, and is then used in combination with rituximab. As of March 2020, venetoclax has also been approved for use in patients who have not yet had any treatment, and is then used in combination with obinutuzumab.

CLL is a rare type of cancer. It typically arises when B cells of the immune system mutate and start multiplying uncontrollably in the blood, bone marrow and lymphatic system. CLL usually progresses slowly and doesn't cause any noticeable symptoms in the early stages. As the disease advances, infections, anemia, severe tiredness and exhaustion are common. Enlarged lymph nodes and an enlarged spleen are also typical signs.

In CLL, the body produces more of a certain protein called BCL-2. This allows more mutated B cells to survive. Venetoclax is used to inhibit BCL-2, stopping the disease from progressing.

Some patients also have specific genetic characteristics: A 17p deletion means that a gene isn't working properly. These patients can't have chemo-immunotherapy.

Venetoclax is an option for use on its own (monotherapy) in:

  • people without a 17p deletion or TP53 mutation if chemo-immunotherapy and treatment with a B-cell receptor inhibitor (such as idelalisib or ibrutinib) wasn't successful, or
  • people with a 17p deletion or TP53 mutation if treatment with a B-cell receptor inhibitor isn't an option or wasn't successful.

In people who have had at least one other treatment, venetoclax can be used in combination with rituximab.

In people who haven't had any treatment, venetoclax can be used in combination with obinutuzumab.

Application

Venetoclax is available in the form of tablets, in three doses (10, 50 and 100 mg). The daily dose starts at 20 mg per day for 7 days. It is then increased once a week until it reaches 400 mg after 5 weeks. The dose can be adjusted if side effects occur or if it's not well tolerated.

Other treatments

For people with CLL who have had treatment before, the following treatment options are available, depending on their previous treatment, genetic characteristics of the cancer, and their general health:

  • the drug ibrutinib or a combination of idelalisib and rituximab,
  • individually tailored chemo-immunotherapy or
  • best supportive care (BSC). This should be tailored to the patient's individual needs, with the aim of relieving symptoms and improving quality of life.

Ibrutinib or specific forms of chemo-immunotherapy are options for the treatment of CLL in people who have not had any previous treatment, depending on the genetic characteristics of the cancer and how well chemo-immunotherapy is tolerated.

Assessment

In 2020, the Institute for Quality and Efficiency in Health Care (IQWiG, Germany) last looked into the advantages and disadvantages of venetoclax (used alone or together with rituximab or obinutuzumab) compared with the standard treatments for people with chronic lymphocytic leukemia.

The manufacturer didn't provide any suitable data with which to do this assessment.

More information

This information summarizes the main results of reviews produced by the Institute for Quality and Efficiency in Health Care (IQWiG, Germany). The reviews were commissioned by the German Federal Joint Committee (G-BA) as part of the “early benefit assessment of medications.” On the basis of the reviews and the hearings received, the G-BA passed a resolution on the added benefit of venetoclax (Venclyxto, Venclexta) when used on its own or in combination with another drug in patients who have already had treatment and in patients who have not had any treatment.

Institute for Quality and Efficiency in Health Care (IQWiG, Germany). Venetoclax (chronic lymphocytic leukaemia) – Benefit assessment according to §35a Social Code Book V. Dossier assessment; Commission A18-81. February 27, 2019. (IQWiG reports; Volume 729).

Institute for Quality and Efficiency in Health Care (IQWiG, Germany). Venetoclax (chronic lymphocytic leukaemia) – Benefit assessment according to §35a Social Code Book V. Dossier assessment; Commission A18-82. February 27, 2019. (IQWiG reports; Volume 730).

Institute for Quality and Efficiency in Health Care (IQWiG, Germany). Venetoclax (chronic lymphocytic leukaemia; combination with rituximab) – Addendum to Commission Auftrag A18-81. Commission A19-35. April 26, 2019. (IQWiG reports; Volume 761).

Institute for Quality and Efficiency in Health Care (IQWiG, Germany). Venetoclax (chronic lymphocytic leukaemia) – Benefit assessment according to §35a Social Code Book V. Dossier assessment; Commission A20-39. July 13, 2020. (IQWiG reports; Volume 946).

Institute for Quality and Efficiency in Health Care (IQWiG, Germany). Venetoclax (chronic lymphocytic leukaemia) – Addendum to Commission A20-39; Commission A20-76. September 25, 2020. (IQWiG reports; Volume 968).

IQWiG health information is written with the aim of helping people understand the advantages and disadvantages of the main treatment options and health care services.

Because IQWiG is a German institute, some of the information provided here is specific to the German health care system. The suitability of any of the described options in an individual case can be determined by talking to a doctor. informedhealth.org can provide support for talks with doctors and other medical professionals, but cannot replace them. We do not offer individual consultations.

Our information is based on the results of good-quality studies. It is written by a team of health care professionals, scientists and editors, and reviewed by external experts. You can find a detailed description of how our health information is produced and updated in our methods.

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Updated on August 13, 2020
Next planned update: 2023

Authors/Publishers:

Institute for Quality and Efficiency in Health Care (IQWiG, Germany)

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