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Evidence-Based Oncology December 2015
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FDA Updates: Oncology
Surabhi Dangi-Garimella, PhD

FDA Updates: Oncology

Surabhi Dangi-Garimella, PhD
Several new FDA approvals and research advances in oncology have the potential for improved outcomes among cancer patients.
The new class of immuno-oncology molecules are definitely keeping up with their promised potential. Nivolumab was approved as first line for treatment-naïve melanoma patients whose tumors express a wild type (WT) BRaf V600.1 The approval follows 5 months after Bristol-Myers Squibb submitted phase 3 results of the Checkmate-066 trial for FDA review.

With overall survival (OS) as the primary endpoint, Checkmate-066 evaluated nivolumab as a single agent in treatment-naïve patients with unresectable or metastatic BRaf WT melanoma. Patients received either nivolumab (n = 210; 3 mg/kg intravenously, once every 2 weeks) or dacarbazine (n = 208; 1000 mg/m2, once every 3 weeks). Progression-free survival (PFS) and objective response rate (ORR) were the secondary endpoints. Interim trial analysis showed a superior OS with nivolumab compared with dacarbazine. Median OS was not reached for nivolumab at the time of analysis; for dacarbazine, it was 10.8 months (95% CI, 9.3-12.1). Nivolumab significantly improved PFS: 5.1 months (95% CI, 3.5-10.8) compared with 2.2 months (95% CI, 2.1-2.4) for patients treated with dacarbazine (Hazard ratio [HR], 0.43; 95% CI, 0.34- 0.56; P <.0001), and ORR was 34% (4% complete response rate [CRR], 30% partial response rate [95% CI, 28-41]) compared with 9% with dacarbazine (1% CRR, 8% partial response rate [95% CI, 5-13]). Nearly 88% of patients had ongoing responses at the time of analysis, about 68% of whom had at least a 6-month response.

Jeffrey S. Weber, MD, PhD, deputy director of the Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center who has been actively involved with Checkmate-066, said, “Advanced melanoma continues to be one of the deadliest and most challenging cancers to treat, and ongoing research in immunooncology from clinical trials like CheckMate -066 shows the potential to provide improved overall survival for newly diagnosed patients with BRAF wild-type metastatic melanoma. This important news means that we now have another new option to offer patients with BRAF wild-type metastatic melanoma.”

Grade 3 and 4 adverse events (AE) were observed in 43% of patients treated with nivolumab, the most common being gamma-glutamyltransferase increase and diarrhea. AE resulted in permanent discontinuation of nivolumab treatment in 7% of patients and dose interruption in 26% of patients. Other documented AE included fatigue, musculoskeletal pain, rash, and pruritis.


1. Bristol-Myers Squibb announces US Food and Drug Administration approval for Opdivo (nivolumab) as a single agent for the treatment of patients with previously untreated BRAF wild-type advanced melanoma [press release]. Princeton, NJ: Bristol- Myers Squibb; November 24, 2015. administration-approval-opdivo-nivolum.

Nivolumab Approved for Third Indication: Advanced Renal Cell Carcinoma

Two months after being granted a Breakthrough Therapy designation by the FDA for metastatic renal cell carcinoma (mRCC), nivolumab was today approved by regulators for treating mRCC patients who have failed a certain type of prior therapy.

The approval1 was based on the results of a phase 3 trial published earlier this month in the New England Journal of Medicine, which compared nivolumab with everolimus in 821 patients with advanced clear-cell renal-cell carcinoma who had received prior treatment with 1 or 2 antiangiogenic agents. The randomized trial assigned patients to receive either an intravenous infusion of nivolumab (3mg/ kg) every 2 weeks or oral everolimus (10 mg tablet, once daily). The secondary endpoints were objective response rate and safety, and the primary trial endpoint was overall survival. The study design allowed dose modification for everolimus but not for nivolumab.

This international study, dubbed Checkmate-025, recruited patients at 146 sites across 24 countries in 5 continents (North and South America, Australia, Asia, and Europe). Only 803 of the 821 patients were treated, with 406 in the nivolumab group and 397 in the everolimus group. By data cutoff in June 2015, 17% (67) of patients in the nivolumab group and 7% (28) in the everolimus group continued to receive treatment. Minimum follow-up period was 14 months and disease progression as the primary reason for discontinuation of treatment (observed in 70% of nivolumab-treated patients and 69% of everolimus-treated patients).

Patients in the nivolumab group had significantly better overall survival (25 months; 95% confidence interval (CI), 21.8 to not estimable) compared with those treated with everolimus (19.6 months; 95% CI, 17.6 to 23.1). Nearly 45% of patients (183 of 410) in the nivolumab cohort died, as opposed to 52% (215 of 411) in the everolimus group. The hazard ratio for all-cause death was 0.73 (98.5% CI, 0.57 to 0.93; P = .002) for nivolumab versus everolimus.

In the press release announcing the drug’s approval, Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, said, “Opdivo [nivolumab] provides an important therapy option for patients with renal cell carcinoma. It is one of few therapies that have demonstrated the ability to extend patients’ survival in treating this disease. Additionally, Opdivo’s extended indication, from melanoma and nonsmall cell lung cancer to renal cell cancer, demonstrates how immune therapies can benefit patients across a wide range of tumors.” EBO


1. FDA approves Opdivo to treat advanced form of kidney cancer [press release]. Silver Spring, MD: FDA; November 23, 2015. medium=email&utm_source=govdelivery.

2. Motzer RJ, Escudier B, McDermott, DF; CheckMate 025 Investigators. Nivolumab versus everolimus in advanced renalcell carcinoma. N Engl J Med. 2015;373(19):1803-1813.

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