Jazz Pharmaceuticals plc, a biopharmaceutical company, announced that the US Food and Drug Administration (FDA) approved a revised label for Vyxeos (daunorubicin and cytarabine) to include a new indication to treat newly-diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC) in pediatric patients aged one year and older.
The approval of Vyxeos for this indication is supported by safety data from two single-arm trials AAML1421, conducted by the Children's Oncology Group (COG) and CPX-MA-1201, conducted by Cincinnati Children's Hospital (CCH) and evidence of effectiveness from an adequate and well-controlled study in adults.
"At Jazz Pharmaceuticals, we believe all patients living with complex conditions deserve solutions, and work diligently to expand the science behind our therapies to ensure the greatest number of patients can benefit from our medicines," said Robert Iannone, M.D., M.S.C.E., executive vice president, research and development and chief medical officer of Jazz Pharmaceuticals. "While pediatric patients represent a relatively small percentage of total AML patients, there is a critical need for more effective therapies in this setting. With the expansion of the Vyxeos label to include the pediatric population, Jazz demonstrates our continued commitment to broadening our cancer research and focusing on the people for whom we can have the greatest impact."
Safety and pharmacokinetics of Vyxeos in children and young adults were established in two clinical studies that enrolled patients with AML or relapsed/refractory hematologic malignancies. Thirty-eight pediatric patients aged one to 21 years of age with AML in first relapse were enrolled in the phase 1/2 AAML1421 study conducted by COG, and 27 patients aged one to 19 years with relapsed/refractory hematologic malignancies were enrolled in the phase 1 CPX-MA-1201 study conducted by CCH. Both studies found no differences in the safety profile based on age. The use of Vyxeos for this indication is supported by evidence of effectiveness from study CPX351-301 in adult patients.
"The expansion of the Vyxeos label to include children is a welcome and necessary advancement in support of some of our most vulnerable patients," said Dr. Edward Anders Kolb, M.D., director of the Center for Cancer and Blood Disorders at Nemours/Alfred I. DuPont Hospital for Children and chair of myeloid disease committee at COG. "Jazz has been a wonderful partner in pediatric drug development and we are grateful for the continued work being done to provide safe and effective therapies for children."
Vyxeos (daunorubicin and cytarabine) is a liposomal combination of daunorubicin, an anthracycline topoisomerase inhibitor, and cytarabine, a nucleoside metabolic inhibitor, that is indicated for the treatment of newly-diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC) in adults and pediatric patients 1 year and older.
Acute myeloid leukemia (AML) is a type of cancer in which the bone marrow makes abnormal myeloblasts (a type of white blood cell), red blood cells, or platelets. It can sometimes spread to other parts of the body including the lymph nodes, liver, spleen, central nervous system (brain and spinal cord), and testicles. AML is a relatively rare disease representing 1.1 percent of all new cancer cases. It is estimated that more than 19,500 people will be diagnosed with AML in the United States this year with the potential for more than 11,000 people to die from the disease.
The median age at diagnosis is 68 years old, with rising age associated with a progressively worsening prognosis.7 AML in children makes up a small portion of the overall AML population (4.5% occurs in patients < 20 years old). Further, t-AML and AML-MRC in pediatric AML are very rare subtypes of this group accompanied by poor prognosis. There is also a reduced tolerance for intensive chemotherapy as patients age. AML has the lowest survival rate of any other form of leukemia. Patients with newly diagnosed therapy-related AML or AML with myelodysplasia-related changes may have a particularly poor prognosis. A hematopoietic stem cell transplant may be a curative treatment option for patients.