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FDA Approves New Maintenance Treatment for AML

By LLS Staff | September 02, 2020

Some promising news to kick off Blood Cancer Awareness Month – on September 1, 2020, the U.S. Food and Drug Administration approved a new oral maintenance treatment for certain adult patients with acute myeloid leukemia (AML).

CC-486 (Onureg®) is now approved for the continued treatment of adult patients with AML who achieved first complete remission (CR) or CR with incomplete blood count recovery (Cri) following intensive induction chemotherapy and who are not able to undergo intensive curative therapy, such as hematopoietic stem cell transplant.

CC-486 is a new oral formulation of azacitidine, an existing therapy that is administered via infusion or injected subcutaneously at a clinic or hospital. This approval establishes an important new maintenance treatment for AML patients in remission that can be taken once daily as a pill.

The approval is based on data from the QUAZAR AML-001 study, which showed that the therapy resulted in a statistically significant and clinically meaningful improvement in overall survival of nearly 10 months, compared to placebo.

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The Leukemia & Lymphoma Society (LLS) helped laid the foundation for this advance, supporting early proof-of-concept work through our research grants portfolio. We continue to lead the charge against AML through our groundbreaking Beat AML Master Clinical Trial – the first collaborative precision medicine clinical trial in a blood cancer – and our investment in the most cutting-edge AML research around the globe, which you can learn more about here.

LLS helped advance 55 of the 65 blood cancer treatment options approved by the U.S. Food and Drug Administration since 2017.