FDA grants priority review to CC-486 for AML maintenance
The FDA granted priority review designation to CC-486 for maintenance treatment of certain adults with acute myeloid leukemia.
The designation applies to use of the agent by patients who achieved complete remission or complete remission with incomplete blood count recovery after induction therapy with or without consolidation treatment, and who are not candidates for or decide not to undergo hematopoietic stem cell transplantation.
The FDA is expected to make a decision on approval of CC-486 (Bristol-Myers Squibb) — an investigational oral hypomethylating agent — by Sept. 3.
The agency granted priority review based on results of the randomized phase 3 QUAZAR-AML-001 study, which included 472 patients with AML who achieved first complete remission or complete remission with incomplete blood count recovery after intensive induction chemotherapy — with or without consolidation — and were ineligible for HSCT.
Researchers assigned patients 1:1 to CC-486 dosed at 300 mg or placebo once daily for 14 days of a 28-day cycle, plus best supportive care. Treatment continued until disease progression or unacceptable toxicity.
OS served as the primary endpoint. Key secondary endpoints included RFS, safety and tolerability, health care resource utilization and patient-reported outcomes.
As Healio previously reported, the study met its primary endpoint.
Patients assigned CC-486 achieved significantly longer median OS (24.7 months vs. 14.8 months; HR = 0.69; 95% CI, 0.55-0.86) and RFS (10.2 months vs. 4.8 months; HR = 0.65; 95% CI, 0.52-0.81). The 1-year relapse rate was 53% in the CC-486 group and 71% in the placebo group.
The most common adverse events reported with CC-486 were gastrointestinal in nature. These included nausea (65% for CC-486 vs. 24% for placebo), vomiting (60% vs. 10%) and diarrhea (50% vs. 22%). Most were grade 1 or grade 2.
“Often, newly diagnosed adult patients with AML achieve a complete response with induction therapy; however, many patients will relapse and experience a poor outcome,” Noah Berkowitz, MD, PhD, senior vice president of global clinical development for hematology at Bristol-Myers Squibb, said in a company-issued press release. “Patients in remission are seeking treatment options that decrease the likelihood of relapse and extend overall survival. [This] acceptance of our submission for CC-486 represents an important step towards a potential new maintenance treatment to address an urgent medical need for [patients with AML], and we look forward to working with the FDA during its review of CC-486.”