Vyxeos™ Receives Positive CHMP Opinion for Treatment of Certain Types of High-Risk Acute Myeloid Leukaemia
"Jazz is committed to bringing new and clinically meaningful treatment options to patients on a global basis, and we now look forward to bringing Vyxeos to adults with AML in the
The Marketing Authorisation Application (MAA) for Vyxeos includes clinical data from five studies, including the pivotal Phase 3 study. Data from the Phase 3 study, which met its primary endpoint, were presented at the
Vyxeos 44 mg/100 mg powder for concentrate for solution for infusion is an advanced liposomal formulation that delivers a fixed-ratio (1:5) of daunorubicin and cytarabine to the bone marrow that has been shown to have synergistic effects at killing leukaemia cells in vitro and in animal models. Vyxeos is the first product developed with the company's proprietary CombiPlex® platform, which enables the design and rapid evaluation of various combinations of therapies. Vyxeos received Orphan Drug Designation by the
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.1 AML cells crowd out healthy cells and move aggressively into the bloodstream to spread cancer to other parts of the body.2 The median age at diagnosis is 68 years old, with rising age associated with a progressively worsening prognosis.3-4 There is also a reduced tolerance for intensive chemotherapy as patients age.5 Patients with t-AML or AML-MRC have few treatment options and some of the lowest survival rates compared to people with other forms of leukaemia.6-7 A hematopoietic stem cell transplant (HSCT) may be a curative treatment option for patients.8
"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 the potential approval and availability of Vyxeos as a treatment option for European patients with 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 regulatory approval process, including the company's ability to obtain approval for Vyxeos in the EU in a timely manner or at all; the manufacture and effective commercialization of Vyxeos in the EU; and other risks and uncertainties affecting the company and its development programs, including those described from time to time under the caption "Risk Factors" and elsewhere in
National Cancer Institute. General Information About Adult Acute Myeloid Leukemia https://www.cancer.gov/types/leukemia/patient/adult-aml-treatment-pdq Accessed June 7, 2017. American Cancer Society. What is Acute Myeloid Leukemia? https://www.cancer.org/cancer/acute-myeloid-leukemia/about/what-is-aml.html Accessed March 20, 2017.
- SEER Stat Facts: AML. 2017.
- Baer MR, et al., Leukemia, 2011 May; 25(5):10.1038/eu.2011.9.
- Klepin HD. Hematology Am Soc Hematol Educ Program. 2014 (1):8-13.
- Pinto AC, et al. Current
Cancer Treatment- Novel Beyond Conventional Approaches. 2011; 693-714.
- Smith SM, et al. Blood. 2003; 102(1):43-52.
- Peccatori, J and Ciceri, F. Haematologica. 2010 Jun; 95(6): 857–859. doi: 10.3324/haematol.2010.023184.
Media Contact: Jacqueline Kirby, Vice President, Corporate Affairs & Government Relations, Ireland +353 1 697 2141, U.S. +1 215 867 4910; Investor Contact: Kathee Littrell, Vice President, Investor Relations, Ireland +353 1 634 7887, U.S. +1 650 496 2717