On October 5, 2017 the National Institute for Health and Care Excellence(NICE) reported a final appraisal determination (FAD) recommending that AbbVie’s VENCLYXTO(venetoclax) is made available to NHS patients with difficult-to-treat types of chronic lymphocyticleukaemia (CLL) via the Cancer Drugs Fund (CDF), providing conditions of the managed accessagreement are followed (Press release, PharmaTimes, OCT 5, 2017, View Source [SID1234520814]). Venetoclax will now be available on the NHS to adult patients in Englandwith CLL in the absence of 17p deletion or TP53 mutation who have failed bothchemo-immunotherapy and a B-cell receptor (BCR) inhibitor. Venetoclax has also beenrecommended for the treatment of adult CLL patients in the presence of 17p deletion or TP53mutation who are either unsuitable for or have failed a BCR inhibitor.2 Please see the NICE websitefor the eligibility criteria: View Source appraisal-determination-document.
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
Today’s recommendation marks the continuation of patient access across the UK, following therecent acceptance of venetoclax for use across NHS Scotland this August.
The immediate inclusion of venetoclax in the Cancer Drugs Fund is a positive step forward forpatients with CLL in England" commented David Innes, Chair of the CLL Support Association. "Accessto new treatment options is vital for patients with challenging forms of CLL, who have a short lifeexpectancy after exhausting current treatment options. We are pleased to see AbbVie and NICEworking together to expedite patient access and are hopeful that this will ultimately translate intolonger-term routine prescribing on the NHS, providing an essential treatment option for those livingwith CLL and their families."
Venetoclax, is a first-in-class, oral, once-daily medicine that selectively inhibits the function of theBCL-2 protein, restoring the body’s ability to trigger cancer cell self-destruction.2 For those patientsliving with CLL requiring treatment, the majority will eventually have their disease recur,3 with one intwo patients failing current treatments facing survival as short as three months.4,5 Venetoclax isbeing developed by AbbVie and Roche. It is jointly commercialised by AbbVie and Genentech, amember of the Roche Group, in the U.S. and by AbbVie outside of the U.S.
Dr Peter Hillmen, Professor of Experimental Haematology and Honorary Consultant Haematologistat Leeds Teaching Hospitals NHS Trust, commented, "Today’s recommendation is great news forpatients with CLL who have failed existing treatments, and provides clinicians with an important newtreatment option. The studies that NICE has assessed to reach this positive decision represent a 4UB 2 milestone in the management of relapsed/refractory CLL. The early clinical data is compelling,showing survival benefits for this challenging group of patients, including some who achievedcomplete remission. I would anticipate that collection of further data through the CDF will confirmthese extremely promising early findings."
CLL affects the blood and immune system and is the most common form of adult leukaemia withalmost 3,500 people affected in the UK each year, with over 3,000 cases in England alone.6,7 Forpeople who develop or harbour gene mutations, such as 17p deletion and/or TP53 mutation,treatment is particularly challenging and these are associated with poorer quality of life and amedian life expectancy of less than two to three years with current standard-of-care regimens.
In a Phase 2 study (M13-982) of 158 patients with relapsed and/or refractory CLL with a 17pdeletion, the overall response rate was 77.2% (122/158) according to investigator assessment.10,11Based on Kaplan-Meier estimations, 86.7% of patients were estimated to be alive following 12months of treatment.11 In a separate Phase 2 two arm study (M14-032) of venetoclax in 64 CLLpatients who relapsed or were refractory to BCR inhibitors (ibrutinib or idelalisib), the primaryendpoint, overall response rate, was 67% and 57% respectively, according to investigatorassessment.11 Venetoclax has also demonstrated early and sustained improvements in fatigue, adebilitating symptom of CLL, with reductions observed at just 4 weeks.
A recent study supports the use of Minimal Residual Disease (MRD) negativity as a prognosticmarker for long-term progression-free survival and as a potential therapeutic goal in CLL. MRDnegativity describes the presence of a small number of leukaemic cells that remain followingtreatment and is defined as <1 CLL cell detectable per 10,000 leukocytes.13,14 In a Phase 2 study inpatients with relapsing and refractory CLL with the del(17p) gene mutation, a high risk prognosticfactor, MRD was used as an exploratory endpoint. Of 158 patients who were treated withvenetoclax, 24% of patients (38/158) achieved MRD negativity in the peripheral blood, including 16patients who were also MRD negative in the bone marrow.
Venetoclax was the first blood cancer medicine to be given positive scientific opinion through theEarly Access to Medicines Scheme (EAMS), following its designation as a Promising InnovativeMedicine (PIM) by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA)designation.
4UB 3As part of AbbVie’s ongoing focus on delivering breakthrough medicines, it has worked with theMHRA and NHSE to provide 50 patients in the UK with early access to venetoclax via EAMS. OnceEAMS ceased, AbbVie made a commitment to providing the treatment free of charge untilreimbursement. Through a combination of EAMS and free of charge supply, approximately 100patients with a high unmet need have benefitted from early access to venetoclax.