Nivolumab Becomes First FDA-Approved Checkpoint Blockade for Kidney Cancer

New hope now exists for patients with advanced kidney cancer. Yesterday, the FDA approved the immunotherapy drug nivolumab (Opdivo^®, Bristol-Myers Squibb) to treat metastatic renal cell carcinoma (RCC)—the most common form of kidney cancer—that has failed to respond to a previous treatment. This breakthrough can be traced all the way back to the research of scientists funded by the Cancer Research Institute (CRI).

Kidney cancer has proven difficult to treat, with only temsirolimus (Torisel^®, Pfizer) and everolimus (Afinitor^®, Novartis) showing benefits in phase III clinical trials. In this most recent study, 821 patients whose advanced kidney cancer did not respond to a therapy designed to stop new blood vessel growth were treated with either nivolumab or everolimus, the current clinical standard.

Patients who received nivolumab had a median overall survival that was over 5 months longer compared to everolimus (25 months versus 19.6 months). The response rate was also over 5 times higher for nivolumab-treated patients (21.5% versus 3.9%) and led to responses lasting nearly twice as long (median duration of 23 months versus 13.7 months). Additionally, half as many serious adverse effects were reported for nivolumab compared to everolimus (19% versus 37%), yet again indicating some of the benefits that immunotherapies often offer over traditional anti-tumor drugs.

Nivolumab represents the first checkpoint inhibitor treatment approved for treatment of advanced kidney cancer. The immunotherapy drug has also been approved for melanoma and lung cancer.

The finding that nivolumab offers clinical success against a third type of cancer reveals the potential of immunotherapies and “demonstrates how [they] can benefit patients across a wide range of tumors,” according to Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research.

The FDA granted priority review status, breakthrough therapy designation, and fast track designation to nivolumab’s application for advanced kidney cancer therapy on November 16. Only one week later, the application received approval, highlighting the FDA’s commitment to making beneficial treatments available to patients as soon as possible.

Nivolumab targets the PD-1 receptor on T cells and acts as a checkpoint inhibitor, “releasing the brakes” on the immune system, and thus enables a stronger anti-tumor immune response. CRI funded research in four laboratories that contributed to our understanding of the PD-1 pathway, and bestowed our highest scientific honor, the 2014 William B. Coley Award, to Lieping Chen, MD, PhD, Gordon Freeman, PhD, Tasuku Honjo, MD, PhD, and Arlene Sharpe, MD, PhD, for their groundbreaking research on PD-1.

Nivolumab, along with pembrolizumab (Keytruda^®, Merck), which also targets PD-1, and ipilimumab (Yervoy^®, Bristol-Myers Squibb), which targets the CTLA-4 receptor, are checkpoint inhibitors that have gone on to win FDA approval. But PD-1 pathway treatments have also shown promise in bladder, head and neck, colorectal, and many other cancer types. We hope that this will be the beginning, and not the end, of PD-1 therapies for a wide spectrum of cancers.