Jazz Pharmaceuticals announced the submission of a Marketing Authorization Application (MAA) to the EMA'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 U.K. 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 III study. Data from the phase III 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.
"Despite numerous advances in the treatment of cancer in Europe, patients with high-risk AML defined as t-AML or AML-MRC have limited treatment options and some of the lowest survival rates compared to people with other forms of AML," said Dr. Nigel Russell, Professor of Haematology, Faculty of Medicine & Health Sciences at the University of Nottingham. "The hematology oncology community has worked tirelessly to better understand the causes and disease pathways associated with AML and we look forward to the arrival of innovative new treatment options."