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FDA Approves Vyxeos for Secondary Acute Myeloid Leukemia in Children

Former TAP Partner


DUBLIN, March 30, 2021 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that the U.S. 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.

LLS Blog Post - LLS applauds every advance for children with blood cancers

The therapy was previously approved in 2017 for adult patients who acquired AML after being treated for a previous cancer or whose AML developed after progressing from a diagnosis of myelodysplastic syndrome. LLS recognized this therapy's potential back in 2009 when it invested in it through its Therapy Acceleration Program® (TAP), which invests in cutting-edge companies working in areas of high unmet need for blood cancer patients. LLS partnered with Celator Pharmaceuticals, which Jazz Pharmaceuticals later acquired, on the clinical development of daunorubicin and cytarabine (Vyxeos®) for adults with AML. 

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