Jazz Pharmaceuticals announced that the National Institute for Health and Care Excellence (NICE) has published a Final Appraisal Determination (FAD) recommending Vyxeos 44 mg/100 mg powder for concentrate for solution for infusion for routine use on the National Health Service (NHS) in England and Wales for the treatment of adults with newly diagnosed, therapy-related acute myeloid leukaemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC)1—two types of secondary AML.
“This is the first new chemotherapy in forty years for adults with specific types of newly diagnosed secondary AML, a particularly aggressive cancer that typically affects older people and has a high mortality rate,” said Dr. Nigel Russell, professor of Haematology, Faculty of Medicine & Health Sciences at the University of Nottingham. “I am pleased that NICE has recognised the value of this medicine for adults with secondary AML. In time, it is expected to become the standard of care for this specific group of older AML patients.”
Patients diagnosed with t-AML or AML-MRC have a very poor prognosis and have the poorest survival of all AML diagnostic subgroups.2,3 Thus they are classified as having high risk disease because of these poor outcomes. In the UK, the expected number of these high-risk AML cases is 680 per year.4 Its incidence increases with age and accounts for approximately 25 percent of all AML cases in the UK.4
“A diagnosis of acute myeloid leukaemia can have a huge emotional impact on the lives of patients, as well as their family and friends,” said Zack Pemberton-Whiteley, patient advocacy director at Leukaemia Care. “We welcome the decision to recommend this treatment for adults with high-risk AML. Leukaemia Care has been working with NICE to enable patients to access this important new medicine, where options have been previously limited.”
“Jazz is delighted that Vyxeos will now be made routinely available on the NHS in England and Wales as people with therapy-related AML or AML with myelodysplasia-related changes have had limited treatment options until now,” said Iain McGill, senior vice president, Europe and Rest of World at Jazz Pharmaceuticals. “We believe that it is a meaningful medicine for patients with this rapidly progressing and life-threatening blood cancer.”
Vyxeos is an advanced liposomal formulation that delivers a synergistic molar ratio of daunorubicin and cytarabine. It is the first chemotherapy to demonstrate a significant overall survival advantage versus the current treatment standard, 7+3 chemotherapy, in a Phase 3 study of older adult patients with newly diagnosed t-AML or AML-MRC,5 and the first chemotherapy treatment option specifically for people who have these types of high-risk AML.
It is an advanced liposomal formulation that has been shown in vitro to deliver a synergistic combination of daunorubicin and cytarabine to leukaemia cells for a prolonged period of time. Based on data in animals, the liposomes accumulate and persist in high concentration in the bone marrow, where they are preferentially taken up intact by leukaemia cells.6 It is the first product developed with the company’s proprietary CombiPlex platform, which enables the consideration and design of various combinations of therapies.
It received Orphan Drug Designation (ODD) by the European Commission in January 2012 with retention of the ODD reaffirmed in July 2018 following assessment by the Committee for Orphan Medicinal Products (COMP), and by the FDA in September 2008 for the treatment of AML. It received Promising Innovative Medicine (PIM) designation from the Medicines and Healthcare Products Regulatory Agency in the United Kingdom.
In August 2018, the European Commission approved it for the treatment of adults with newly diagnosed t-AML or AML-MRC in all European Union Member States, as well as Iceland, Norway, and Liechtenstein.
1. NICE Final Appraisal Document Vyxeos November 2018 www.nice.org.uk
2. Roman E et al. Myeloid malignancies in the real-world: Occurrence, progression and survival in the UK’s population-based Haematological Malignancy Research Network. 2004-2015. Cancer Epidemiology 2016; 42:186-198.
3. Boddu P et al. Treated Secondary Acute Myeloid Leukemia: A Distinct High-Risk Subset of AML with Adverse Prognosis. The American Society of Hematology. 2017; 1(17)
4. HMRN Incidence. AML. Available at: https://www.hmrn.org/statistics/incidence Last accessed October 2018
5. Lancet JE et al. CPX-351 (cytarabine and daunorubicin) Liposome for Injection Versus Conventional Cytarabine Plus Daunorubicin in Older Patients with Newly Diagnosed Secondary Acute Myeloid Leukemia. J Clin Oncol. 2018; 36(26):2684-2692.
6. Vyxeos Summary of Characteristics Available at https://www.medicines.org.uk/emc/product/9442
(Source: Jazz Pharmaceuticals plc)