On April 3, 2017 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported the completion on March 31, 2017 of a rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of Vyxeos (cytarabine and daunorubicin) liposome for injection, an investigational treatment for acute myeloid leukemia (AML), a rapidly progressing and life-threatening blood cancer (Press release, Jazz Pharmaceuticals, APR 3, 2017, View Source [SID1234518440]). 1 The company has requested a priority review for the Vyxeos NDA, which, if granted, would accelerate the expected timing of the FDA’s review. Schedule your 30 min Free 1stOncology Demo! Vyxeos utilizes CombiPlex technology, which encapsulates a fixed ratio of drugs in a nano-scale delivery complex. Vyxeos received Breakthrough Therapy Designation from the FDA in May 2016 for the treatment of adults with therapy-related AML or AML with myelodysplasia-related changes. Vyxeos was also granted Fast Track Designation for the treatment of elderly patients with secondary AML by the FDA, and Orphan Drug Designation by the FDA and the European Commission for the treatment of AML. The FDA grants Breakthrough Therapy Designation to expedite the development and review of new medicines that are intended to treat serious or life-threatening diseases when the clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on at least one clinically significant endpoint.
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"The completion of our NDA submission for Vyxeos marks a significant milestone for the company as we continue to advance our pipeline of products for hematology and oncology diseases with high unmet medical needs," said Karen Smith, M.D., Ph.D., global head of research and development and chief medical officer at Jazz Pharmaceuticals. "We look forward to working closely with the FDA toward the goal of obtaining approval of Vyxeos for patients with AML as little has changed in the pharmacologic treatment of AML in more than 40 years."
The NDA submission includes clinical data from five studies, including the pivotal Phase 3 study. Data from the Phase 3 study, which met its primary endpoint, were previously presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June 2016.
About Vyxeos (CPX-351)
Vyxeos (cytarabine and daunorubicin) liposome for injection, or CPX-351, is an investigational product being evaluated for the treatment of AML, and is a combination of cytarabine and daunorubicin encapsulated within a nano-scale liposome at a 5:1 molar ratio. The proposed trade name, Vyxeos, is conditionally approved by the FDA and is subject to confirmation upon approval of the NDA.
About Acute Myeloid Leukemia
Acute Myeloid Leukemia (AML) is a rapidly progressing and life-threatening blood cancer that rises in frequency with age.1 The American Cancer Society estimates that there will be approximately 21,380 new cases of AML and 10,590 deaths from AML in the United States in 2017.1 The median age at diagnosis is 67 and with rising age there is progressive worsening of prognosis.1,2 Advancing age is associated with increasing risk of specific chromosomal/mutational changes and risk of pre-malignant marrow disorders, which give rise to more aggressive and less responsive forms of AML.3,4 As patients age there is also reduced tolerance for intensive chemotherapy.5 As a consequence, advances in supportive care, intensive chemotherapy and bone marrow transplantation have primarily benefitted younger patients.3,5 The five-year survival rate has not improved in older patients despite of 40 years of research.5,6