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FDA Approves Vyxeos for Adults with Poor Prognosis AML.

The U.S. Food and Drug Administration granted regular approval to a liposome-encapsulated combination of daunorubicin and cytarabine for the treatment of adults with newly-diagnosed therapy-related AML (t-AML) or AML with myelodysplasia-related changes (AML-MRC), two types of AML having a poor prognosis.

This is the first FDA-approved treatment specifically for patients with t-AML or AML-MRC.

Approval was based on a multicenter, open-label, active-controlled trial comparing Vyxeos to a standard combination of daunorubicin and cytarabine (7+3) in 309 patients 60-75 years of age with newly-diagnosed t-AML or AML-MRC. Vyxeos demonstrated an estimated median overall survival of 9.6 months compared with 5.9 months for the 7+3 control.

Results from a recent phase III clinical trial have demonstrated an improved survival benefit with the use of Vyxeos (CPX-351) compared with the standard chemotherapy combination consisting of cytarabine and daunorubicin (7+3). These results will be submitted for presentation at the upcoming 2016 annual meeting of the American Society of Clinical Oncology (ASCO).

Acute myeloid leukemia (AML) is diagnosed in approximately 20,000 individuals each year in the United States. It is an aggressive leukemia, with the lowest survival rates of any acute leukemias.

AML prevents certain immune cells from developing properly, leaving them in immature stages. These cancerous cells, referred to as “blasts”, are not able to fight infection as intended, and rapidly accumulate in the body. This crowds out other blood cells so that they are not able to carry out their essential functions.

The cornerstone of standard therapy for AML is chemotherapy, and has remained essentially unchanged in the past 25 years, demonstrating a clear need for novel strategies.

One commonly used treatment for the elderly in AML includes the combination of the chemotherapy agents, cytarabine and daunorubicin. This combination is referred to as 7+3 in reference of the ratios of the drugs used together.

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However, researchers recently demonstrated that a ratio of 5 to 1, with the same chemotherapy agents, provides improved anti-cancer effects compared to the standard ratio.

Vyxeos is a drug that is in the last phases of clinical trials prior to the submission of approval to the United States Food and Drug Administration (FDA). It is comprised of cytarabine and daunorubicin in the 5 to 1 ratio.

A phase III clinical trial was recently conducted to directly compare Vyxeos to the standard 7+3 combination of cytarabine plus daunorubicin for elderly patients with AML who were at a high risk of developing a disease recurrence. In the trial, one group of patients was treated with Vyxeos, and a second group was treated with the standard 7+3 combination. Results were directly compared between the two groups of patients.

  • Median overall survival was improved by 3.6 months for patients treated with Vyxeos (nearly 10 months), compared to those treated with 7+3 (nearly 6 months).
  • 12 months following initiation of treatment, 41.5% of patients treated with Vyxeos were still alive, compared with only 27.6% of patients treated with 7+3.
  • 24 months following initiation of treatment, 31.1% of patients treated with Vyxeos were still alive, compared with only 12.3% treated with 7+3.
  • The rate of serious side effects was comparable between the two treatment groups.

CPX-351 Receives Fast Track Designation for Secondary Acute Myeloid Leukemia in Elderly Patients

The U. S. Food and Drug Administration (FDA) has granted Fast Track designation to the investigational drug CPX-351, a liposomal formulation of cytarabine-daunorubicin, for the treatment of elderly patients with secondary acute myeloid leukemia.

Acute myeloid leukemia (AML) is a cancer of the bone marrow (spongy portion found in the middle of bones) and blood characterized by the rapid, uncontrolled growth of immature white blood cells known as myelocytes. The disease is more common in adults than in children; the average age at diagnosis is over 65. Treatment of elderly patients with AML remains unsatisfactory, and most elderly patients die within a months of diagnosis.

CPX-351 is a liposomal formulation of cytarabine and daunorubicin in a 5:1 ratio designed to maximize anti-tumor synergy. CPX-351 accumulates within bone marrow with preferential uptake of liposomes by leukemia cells followed by intracellular release of encapsulated drug. CPX-351 has been granted orphan drug status by the U.S. FDA for the treatment of AML.

Fast Track designation from the FDA was designed to facilitate the development and expedite the review of drugs that demonstrate the potential to address an unmet medical need for a serious or life-threatening condition.

A phase III study comparing CPX-351 to the current standard of care has recently completed enrollment. Preliminary data from this clinical trial are expected to be available in the second quarter of this year, and overall survival data, which is the primary endpoint of the study, is expected early in 2016.

The most common adverse reactions occurring in greater than 25% of patients treated with Vyxeos were hemorrhage events, febrile neutropenia, rash, edema, nausea, mucositis, diarrhea, constipation, musculoskeletal pain, fatigue, abdominal pain, dyspnea, headache, cough, decreased appetite, arrhythmia, pneumonia, bacteremia, chills, sleep disorders and vomiting.

Reference: Celator Pharmaceuticals, Inc. Celator Announces Phase 3 Trial for VYXEOS™ (CPX-351) in Patients with High-Risk Acute Myeloid Leukemia Demonstrates Statistically Significant Improvement in Overall Survival. Available at: . Accessed March 15, 2016.

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