Vyxeos Granted FDA Breakthrough Therapy Designation
Celator Pharmaceuticals has announced that VYXEOS (also known as CPX-351), has been granted Breakthrough Therapy designation. VYXEOS is an investigational product in development as a treatment for acute myeloid leukemia (AML) and other blood cancers.
Breakthrough therapy is a United States Food and Drug Administration designation that expedites drug development, and can be assigned to a drug if “it as a drug which is intended alone or in combination with one or more other drugs to treat a serious or life threatening disease or condition” and if the preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.”
The VYXEOS Breakthrough Therapy designation is based on positive results from the pivotal Phase 3 clinical trial in older patients with previously untreated high-risk (secondary) AML.
The designation is for the treatment of adults with therapy-related AML (t-AML) or AML with myelodysplasia-related changes (AML-MRC). This designation includes the patient populations enrolled in the Phase 3 clinical trial.
Adult acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. This type of cancer usually gets worse quickly if it is not treated. It is the most common type of acute leukemia in adults. AML is also called acute myelogenous leukemia, acute myeloblastic leukemia, acute granulocytic leukemia, and acute nonlymphocytic leukemia.
VYXEOS is Celator’s nano-scale liposomal formulation of cytarabine and daunorubicin. The company’s patented delivery system maintains molar ratios of the drugs at the tumor site, unlike other regimes which have their ratios change due to things such as solubility, biological responses, and antagonistic effects.
VYXEOS was granted orphan drug status for the treatment of AML by the FDA and the European Commission. VYXEOS was also granted Fast Track designation for the treatment of elderly patients with secondary AML by the FDA.
“We are very happy the FDA granted Breakthrough Therapy designation for VYXEOS,” said Scott Jackson, Celator’s CEO. “The breadth of the designation, which includes all adults with t-AML and AML-MRC, is encouraging as AML patients are in need of advancements in treatment. We look forward to working with the FDA to bring VYXEOS to patients as quickly as possible.”
Celator plans to submit a New Drug Application (NDA) to the FDA by the end of the third quarter of 2016.