The U.S. Food and Drug Administration (FDA) has accepted Bristol Myers Squibb’s application for CC-486 as a maintenance treatment for adults with acute myeloid leukemia (AML) who achieved complete remission — with or without complete blood count recovery — after induction therapy, and who are not candidates for a stem cell transplant.
The FDA also gave priority review to the application. This designation intends to speed the review process of treatments that, if approved, would significantly improve the safety or effectiveness of treating, diagnosing, or preventing a serious condition.
An approval decision is expected on or about Sept. 3.
“Today’s acceptance of our submission for CC-486 represents an important step towards a potential new maintenance treatment to address an urgent medical need for AML patients and we look forward to working with the FDA during its review of CC-486,” Noah Berkowitz, MD, PhD, senior vice president, global clinical development, hematology at Bristol Myers Squibb, said in a press release.
CC-486 is an oral formulation of azacitidine — a chemotherapy agent that acts at the DNA level to restore the normal expression of genes often ‘turned off’ by cancer cells to allow their uncontrolled proliferation and survival. CC-486 works to lower the number of abnormal blood cells and to control cell growth.
The approval request was based on results from the Phase 3 QUAZAR AML-001 trial (NCT01757535), which evaluated the efficacy and safety of CC-486 as a maintenance therapy for AML patients in complete remission after intensive first-line induction chemotherapy, with or without consolidation chemotherapy, and who were ineligible for a hematopoietic (blood-forming) stem cell transplant.
At the time of the analysis, 472 patients were randomly assigned to receive 300 mg of CC-486 or placebo orally, once daily in a two-weeks-on, two-weeks-off schedule.
Results showed that CC-486 significantly extended overall survival to 24.7 months, compared to 14.8 months in those given placebo. This represented a 31% reduction in the risk of death.
The study also met its endpoint of relapse-free survival, with CC-486 reducing the risk of relapse of death by 35%, and preserved health-related quality of life compared to a placebo.
The most common severe or life-threatening adverse events were low neutrophil (a type of white blood cell) numbers, anemia, and low platelet counts. Serious adverse events, which were mainly infections, were more common with CC-486 than placebo. More patients on CC-486 stopped taking the treatment due to adverse events (13% vs. 4% for placebo).
However, patients assigned CC-486 remained on treatment for longer than those on placebo (12 cycles versus 6 cycles), supporting the treatment’s benefits and tolerability.
“Often, newly diagnosed adult patients with AML achieve a complete response with induction therapy, however many patients will relapse and experience a poor outcome. Patients in remission are seeking treatment options that decrease the likelihood of relapse and extend overall survival,” Berkowitz said.
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