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, the drug is also available to this group of patients in combination with rituximab.

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 certain part of a gene is missing, while a TP53 mutation means that a gene is defective. 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.

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

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.

Assessment

In 2019, the Institute for Quality and Efficiency in Health Care (IQWiG, Germany) looked into the advantages and disadvantages of venetoclax (used alone or together with rituximab) compared with the standard treatments for people with chronic lymphocytic leukemia.

The manufacturer didn't provide any suitable data with which to answer this question.

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 rituximab.