NICE Recommends VENCLYXTO®▼(Venetoclax) Combination in Certain Patients with the Aggressive Blood Cancer Acute Myeloid Leukaemia (AML) 

NICE Recommends VENCLYXTO^®▼(Venetoclax) Combination in Certain Patients with the Aggressive Blood Cancer Acute Myeloid Leukaemia (AML) 

• Venetoclax in combination with azacitidine will be immediately available to untreated adult patients with AML unable to receive intensive chemotherapy (IC) ¹

• The combination has demonstrated ability to offer these patients significantly improved overall survival, increased rates of rapid and durable remission and blood transfusion independence, compared with treatment with azacitidine alone²

• Clinical experts describe the combination as the greatest advance in 30 years³ for those unable to receive IC for this aggressive, difficult-to-treat blood cancer, which has a low survival rate⁴

Maidenhead, UK, 16 December 2021 – The National Institute for Health and Care Excellence (NICE) has today published a positive Final Appraisal Document recommending routine NHS use of VENCLYXTO^® (venetoclax) with azacitidine for the treatment of AML.¹ Venetoclax with azacitidine is recommended, within its marketing authorisation, as an option for untreated AML in adults when IC is unsuitable.¹ The decision means these patients will now benefit from a treatment offering significantly improved overall survival, increased rates of rapid and durable remission and blood transfusion independence, compared with treatment using azacitidine alone.^1,2

AML is an aggressive and difficult-to-treat blood cancer with a low survival rate.⁴ Approximately 3,200 people are diagnosed with AML in the UK every year, which equates to more than eight new cases every day. ⁵ The disease predominately affects older people and around 40% of those newly diagnosed are unable to tolerate the standard first-line treatment, IC, due to fitness or presence of other conditions. ^1,6 The past three decades have seen few advances in the treatment of AML. ⁷ With limited treatment options, the prognosis for these patients has remained poor. Median survival is just 6–10 months, during which patients typically require hospitalisation and frequent blood transfusions impacting their quality of life. ^8,9

Venetoclax is an oral once-daily treatment, which works by blocking the action of the B-cell lymphoma-2 (BCL-2) protein, the presence of which helps cancer cells survive. ¹⁰ As it can be taken at home and reduces dependency on blood transfusions, treatment with venetoclax was considered by patient and clinical experts to likely mean fewer hospital visits for these clinically vulnerable patients, who are more susceptible to coronavirus and other infections. ¹¹ 

Zack Pemberton-Whiteley, chief executive of Leukaemia Care, said ‘AML is a rapidly progressing, life-threatening disease, which has a significant emotional and practical burden on patients and their loved ones. Advances in treatment have been slow, especially for this group of patients. The availability of a new effective and more tolerable treatment, which can be administered largely at home, is very welcome news’. 

Commenting on the decision, Professor Charlie Craddock, academic director of the Centre for Clinical Haematology, Queen Elizabeth Hospital, Birmingham and professor of haemato-oncology, at the University of Birmingham: ‘Venetoclax plus azacitidine is the most important advance in the treatment of AML for patients not suitable for intensive chemotherapy in over 30 years and represents a step change in therapy for this common blood cancer’.

Belinda Byrne, medical director, AbbVie UK said, ‘This is a disease with an urgent need for effective new treatments. Routine availability within the NHS of the venetoclax with azacitidine combination will give patients back precious time with families and improve their quality of life. We are delighted with this recommendation from NICE and commend the committee for its robust and timely consideration of the evidence’.

Venetoclax is being developed by AbbVie and Roche. It is jointly commercialised by AbbVie and Genentech, a member of the Roche Group, in the U.S. and by AbbVie outside of the U.S.

- Ends -

For the venetoclax Summary of Product Characteristics, please visit: https://www.medicines.org.uk/emc/medicine/32650.

▼ Adverse events should be reported. Reporting forms and information can be found at www.mhra.gov.uk/yellowcard. Adverse events should also be reported to AbbVie UK Ltd.
Please contact GBPV@abbvie.com.

NOTES TO EDITORS:

About the National Institute for Health and Care Excellence (NICE) recommendation

The Final Appraisal Document published by NICE today states that venetoclax with azacitidine is recommended, within its marketing authorisation, as an option for untreated acute myeloid leukaemia in adults when intensive chemotherapy is unsuitable. It is recommended only if the company provides venetoclax according to the commercial arrangement.¹

About venetoclax

Venetoclax is a first-in-class medicine that selectively binds and inhibits the B-cell lymphoma 2 (BCL-2) protein. In some blood cancers, BCL-2 prevents cancer cells from undergoing their natural death or self-destruction process, called apoptosis. Venetoclax targets the BCL-2 protein and works to help restore the process of apoptosis.¹⁰ Venetoclax in combination with a hypomethylating agent received marketing authorisation from the Medicines and Healthcare products Regulatory Agency (MHRA) in May 2021 for the treatment of newly diagnosed adults with acute myeloid leukaemia who are ineligible for intensive chemotherapy.¹⁰

VIALE-A Phase 3 trial ²

The recommendation by NICE is based on results from the Phase 3 double-blind, placebo-controlled VIALE-A trial. VIALE-A evaluated the efficacy and safety of venetoclax in combination with azacitidine in patients with newly diagnosed acute myeloid leukaemia who were ineligible for intensive chemotherapy. The study met its primary endpoint of statistically significant improvement of overall survival (OS). OS was 14.7 months for the venetoclax plus azacitidine arm (n=286) versus 9.6 months in the placebo plus azacitidine arm (n=144) (p<0.001). The study also met secondary endpoints, including but not limited to; a composite complete remission rate of 66.4% in the venetoclax plus azacitidine arm vs. 28.3% in the placebo plus azacitidine arm (p<0.001) and greater platelet and red cell transfusion independence with venetoclax plus azacitidine vs. placebo plus azacitidine (59.8% vs. 35.2% and 68.5% and 49.7%, respectively; p<0.001).

Adverse events

In the VIALE-A trial the safety profile of venetoclax plus azacitidine was consistent with the known side effect profiles of both agents, and adverse events (AEs) were consistent with expectations for an older AML population. The most commonly occurring adverse reactions (≥20%) of any grade in patients receiving venetoclax in combination with azacitidine were thrombocytopenia, neutropenia, febrile neutropenia, nausea, diarrhoea, vomiting, anaemia, fatigue, pneumonia, hypokalaemia and decreased appetite.¹⁰ The most frequently reported serious AEs, reported in at least 5% of patients in either treatment arm were febrile neutropenia, pneumonia, sepsis and haemorrhage.¹⁰

About AbbVie in oncology

At AbbVie, we are committed to transforming standards of care for multiple blood cancers while advancing a dynamic pipeline of investigational therapies across a range of cancer types. Our dedicated and experienced team joins forces with innovative partners to accelerate the delivery of potentially breakthrough medicines. We are evaluating more than 20 investigational medicines in over 300 clinical trials across some of the world’s most widespread and debilitating cancers. As we work to have a remarkable impact on people’s lives, we are committed to exploring solutions to help patients obtain access to our cancer medicines. For more information, please visit http://www.abbvie.co.uk.

About AbbVie

AbbVie’s mission is to discover and deliver innovative medicines that solve serious health issues today and address the medical challenges of tomorrow. We strive to have a remarkable impact on people's lives across several key therapeutic areas: immunology, oncology, neuroscience, eye care, virology, women’s health and gastroenterology, in addition to products and services across its Allergan Aesthetics portfolio. For more information about AbbVie, please visit us at www.abbvie.co.uk. Follow @abbvieuk on Twitter or YouTube.

References

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chemotherapy is unsuitable.  Committee Papers. Available from: https://www.nice.org.uk/guidance/gid ta10478/documents/committee-papers [Last accessed: December 2021].

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