Two regimens receive priority review
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The FDA granted priority review to two cancer treatment regimens.
CC-486 (Bristol-Myers Squibb) received priority review 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 — 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. All patients 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 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.
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) than those assigned placebo. 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 toward 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.”
The FDA also granted priority review to nivolumab (Opdivo, Bristol-Myers Squibb) plus ipilimumab (Yervoy, Bristol-Myers Squibb) combined with a limited chemotherapy course as first-line treatment for patients with metastatic or recurrent non-small cell lung cancer.
The designation applies to use of the regimen for patients with no EGFR or ALK genomic tumor aberrations.
Nivolumab is an anti-PD-1 antibody, and ipilimumab is an anti-CTLA-4 antibody. The combination is approved in the United States for treatment of certain patients with melanoma, renal cell carcinoma, colorectal cancer and hepatocellular carcinoma.
The FDA granted priority review to the lung cancer indication based on results of the randomized phase 3 CheckMate-9LA trial, which assessed the combination as first-line treatment for patients with metastatic NSCLC regardless of PD-L1 expression or histology.
Researchers assigned patients in the experimental treatment group to nivolumab dosed at 360 mg every 3 weeks plus ipilimumab dosed at 1 mg/kg every 6 weeks, combined with two cycles of chemotherapy. Treatment continued for up to 2 years or until disease progression or unacceptable toxicity.
Patients in the control group received up to four cycles of chemotherapy alone, followed by optional pemetrexed maintenance therapy if eligible until disease progression or toxicity.
The study met its primary endpoint of improved OS in the intent-to-treat population. The FDA is expected to make a decision on approval by Aug. 6.
“Despite treatment advances, there remains a serious unmet need for additional innovative treatment options for [patients with lung cancer],” Sabine Maier, MD, development lead for thoracic cancers at Bristol-Myers Squibb, said in a company-issued press release. “We look forward to working with regulatory authorities to bring the first and only dual immunotherapy plus limited chemotherapy regimen to patients as soon as possible.”