Venetoclax (Venclyxto™) Infosheet

This Horizons Infosheet contains information on venetoclax, a drug being investigated for the treatment of myeloma.

Venetoclax (also known as Venclyxto™) is a new type of drug being investigated for the treatment of myeloma. It belongs to a class of drugs known as pro-survival inhibitors, which work by accelerating cancer cell death.

Venetoclax is an oral drug, given as a tablet. It is currently being investigated in Phase III clinical trials as a once-daily treatment of 800mg in myeloma.

The most commonly seen side effects of venetoclax in myeloma trials included diarrhoea, constipation, nausea, anaemia,
fatigue, neutropenia (low level of neutrophils, a type of white blood cell) and thrombocytopenia (low level of platelets).

In 2016 venetoclax was granted “orphan drug designation” by the European Medicines Agency (EMA) for myeloma.
This means that the EMA will offer the drug company certain financial incentives throughout the development and  licensing process in order to enable venetoclax to become available to patients sooner. Therefore, venetoclax is not
currently licensed for use in myeloma in the UK and is only accessible to patients as part of a clinical trial.

For an up-to-date list of UK clinical trials involving venetoclax, visit the Myeloma Trial Finder on

Venetoclax continues to be studied in different patient groups and in different combinations, having recently entered Phase III trials in the UK in combination with bortezomib and dexamethasone in relapsed and/or refractory myeloma

The information in the Horizons Infosheet is not meant to replace the advice of your medical team. They are the people to ask if you have questions about your individual situation. All Myeloma UK publications are extensively reviewed by patients and healthcare professionals prior to publication.