Jazz Pharmaceuticals announced the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) for Vyxeos (daunorubicin and cytarabine) powder for concentrate for infusion to treat adults with high-risk acute myeloid leukemia (AML) defined as therapy-related AML (t-AML) or AML with myelodysplasia-related changes (AML-MRC).
The CHMP granted Vyxeos accelerated assessment, which is designed to reduce the review timeline for products of major interest for public health and therapeutic innovation. Vyxeos also received Promising Innovative Medicine (PIM) designation from the Medicines and Healthcare Products Regulatory Agency in the United Kingdom. A PIM designation is an early indication that a medicinal product is a promising candidate for the Early Access to Medicines Scheme (EAMS), intended for the treatment, diagnosis or prevention of a life-threatening or seriously debilitating condition with the potential to address an unmet medical need.
"If approved, Vyxeos will become the first new chemotherapy treatment option specifically for European patients with therapy-related AML or AML with myelodysplasia-related changes," said Karen Smith, M.D., Ph.D., executive vice president, research and development and chief medical officer at Jazz Pharmaceuticals. "We are passionate about bringing a new treatment option for high-risk AML to the appropriate patients in the EU as quickly as possible and look forward to working with the CHMP during this review process."
The MAA for Vyxeos includes clinical data from five studies, including the pivotal Phase 3 study. Data from the Phase 3 study, which demonstrated a statistically significant improvement in overall survival for Vyxeos versus standard of care, were presented at the American Society of Clinical Oncology Annual Meeting in June 2016.
Vyxeos daunorubicin 2.2 mg/mL and cytarabine 5 mg/mL powder for concentrate 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 leukemia 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 European Commission in January 2012 and by the U.S. Food and Drug Administration (FDA) in September 2008 for the treatment of AML. Vyxeos received U.S. FDA approval on August 3, 2017 for the treatment of adults with newly-diagnosed t-AML or AML-MRC.
Acute myeloid leukemia (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 aggressively into the bloodstream to spread cancer to other parts of the body. The median age at diagnosis is 68 years old, with rising age associated with a progressively worsening prognosis. There is also a reduced tolerance for intensive chemotherapy as patients age. 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 leukemia. A hematopoietic stem cell transplant (HSCT) may be a curative treatment option for patients.