Jazz Pharmaceuticals Initiates Rolling NDA Submission for Vyxeos (CPX-351) Expects to Complete NDA Submission by Early 2017

On October 3, 2016 Jazz Pharmaceuticals plc (Nasdaq; JAZZ) reported the initiation of a rolling submission of a New Drug Application (NDA) to the United States (U.S.) Food and Drug Administration (FDA) on September 30, 2016, seeking marketing approval of Vyxeos (cytarabine and daunorubicin liposome injection), an investigational agent for the treatment of acute myeloid leukemia (AML) (Press release, Jazz Pharmaceuticals, OCT 3, 2016, View Source [SID:SID1234515536]). The company expects to complete the submission of the NDA by early 2017, and will request a priority review.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Our initiation of the rolling NDA submission for Vyxeos brings us closer to an important goal of providing a new treatment option for patients with acute myeloid leukemia, a devastating and life-threatening disease," said Karen Smith M.D., Ph.D., global head of research and development and chief medical officer at Jazz Pharmaceuticals. "AML represents a disease area within the hematological cancers, where there remains a significant unmet medical need. Vyxeos, if approved, would be the first new treatment demonstrating a significant advancement in treating AML in over 20 years."

Vyxeos was granted Breakthrough Therapy Designation in May 2016 for the treatment of adults with therapy-related AML or AML with myelodysplasia-related changes. 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. The Breakthrough Therapy designation allows a company to submit individual sections of its NDA for review by the FDA as they are completed rather than waiting until the entire application is complete before it can be submitted and reviewed.

Celator Pharmaceuticals, Inc. completed and announced the results of its Phase 3 trial evaluating Vyxeos in patients with high-risk AML in March 2016. Jazz Pharmaceuticals completed the acquisition of Celator Pharmaceuticals in July 2016.

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 19,950 new cases of AML and 10,430 deaths from AML in the U.S. in 2016.2 In the European Union, the number of new cases is estimated to be 20,100 in 2016.3

The median age at diagnosis is 67 and with rising age there is progressive worsening of prognosis.1,4 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.5,6 As patients age there is also reduced tolerance for intensive chemotherapy.7 As a consequence, advances in supportive care, intensive chemotherapy, and bone marrow transplantation have primarily benefitted younger patients with approximately one third of patients 18-60 years of age achieving cure.5,7 Older patients have not achieved higher rates of cure or improved upon a 5-year survival rate of 10-20% in spite of 40 years of research. 7, 8

About Vyxeos

Vyxeos (cytarabine and daunorubicin liposome injection), or CPX-351, is a combination of cytarabine and daunorubicin encapsulated within a nano-scale liposome at a 5:1 molar ratio. Vyxeos was granted orphan drug status by the FDA and the European Commission for the treatment of acute myeloid leukemia. Vyxeos was granted Breakthrough Therapy Designation for the treatment of adults with therapy-related AML or AML with myelodysplasia-related changes and was also granted Fast Track designation by the FDA for the treatment of older patients with secondary AML.