National Institute for Health and Care Excellence (NICE) Recommends Jazz Pharmaceuticals' Vyxeos® (Daunorubicin and Cytarabine) for Adults with Specific Types of Secondary Acute Myeloid Leukaemia (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
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
"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
"Jazz is delighted that Vyxeos will now be made routinely available on the NHS in
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.
Notes to Editors
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
View the Summary of Product Characteristics here.
The CombiPlex proprietary technology enables the design and rapid evaluation of various combinations of therapies to deliver enhanced anti-cancer activity. The CombiPlex technology seeks to identify the most synergistic ratio of drugs in vitro and fix this ratio in a nano-scale delivery complex that maintains the synergistic combination after administration. CombiPlex utilizes two proprietary nano-scale delivery platforms: liposomes to control the release and distribution of water-soluble drugs and drugs that are both water- and fat-soluble (amphipathic), and nanoparticles to control the release and distribution of non-water-soluble (hydrophobic) drugs.
Acute myeloid leukaemia (AML) is a blood cancer that begins in the bone marrow, which produces most of the body's new blood cells. AML cells crowd out healthy cells and move into the bloodstream to spread cancer to other parts of the body.7 The median age at diagnosis is 72 years old, with rising age associated with a progressively worsening prognosis.4 There is also a reduced tolerance for intensive chemotherapy as patients age.8 Patients with t-AML or AML-MRC have the lowest survival rates compared with people with other forms of leukaemia.2,3 A haematopoietic stem cell transplant (HSCT) may be a curative treatment option for patients.9
"Safe Harbor" Statement under the Private Securities Litigation Reform Act of 1995
This press release contains forward-looking statements, including, but not limited to, statements related to Vyxeos becoming the standard of care for patients with newly diagnosed t-AML or AML-MRC, and other statements that are not historical facts. These forward-looking statements are based on the company's current plans, objectives, estimates, expectations and intentions and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with: the company's ability to effectively commercialize Vyxeos; delays or problems in the supply or manufacture of Vyxeos; maintaining appropriate pricing and reimbursement for Vyxeos; complying with applicable regulatory requirements; and other risks and uncertainties affecting the company, including those described from time to time under the caption "Risk Factors" and elsewhere in
1. NICE Final Appraisal Document Vyxeos
2. Roman E et al. Myeloid malignancies in the real-world: Occurrence, progression and survival in the
3. Boddu P et al. Treated Secondary Acute Myeloid Leukemia: A Distinct High-Risk Subset of AML with Adverse Prognosis.
4. HMRN Incidence. AML. Available at: https://www.hmrn.org/statistics/incidence Last accessed
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 Last accessed
8. Klepin HD. Hematology Am Soc Hematol Educ Program. 2014; (1):8-13
9. Peccatori J and Ciceri F. Haematologica. 2010; 95(6): 857–859. doi: 10.3324/haematol. 2010.023184.
Date of preparation:
Job bag number: EURW-UKVYX-0058
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