FDA Approves Second PD-1 Inhibitor, Nivolumab, for Melanoma

The FDA approved nivolumab for the treatment of patients with unresectable or metastatic melanoma.

The US Food and Drug Administration (FDA) has granted nivolumab (Opdivo), a PD-1 pathway inhibitor, accelerated approval for the treatment of patients with unresectable or metastatic melanoma who no longer respond to other treatments. The drug has been granted breakthrough designation, priority review and orphan product designation.

Nivolumab is the seventh new drug approved for the treatment of melanoma since 2011, including pembrolizumab, another PD-1 inhibitor approved in September.

“The continued development and approval of novel therapies based on our increasing understanding of tumor immunology and molecular pathways are changing the treatment paradigm for serious and life-threatening diseases,” said Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, in a prepared statement.

Nivoluamb is approved for the treatment of patients who have been previously treated with the anti-CTLA-4 inhibitor ipilimumab and, for patients with melanoma with BRAF V600 mutations who have progressed after treatment with ipilimumab and a BRAF inhibitor.

The approval of nivolumab was based on data reported at the 2014 ESMO Congress that examined the drug in patients with advanced melanoma who had progressed on or after anti-CTLA-4 therapy or treatment with a BRAF inhibitor. Patients were randomly assigned 2:1 to treatment with nivolumab or investigator’s choice chemotherapy.

The overall response rate was assessed in 120 patients assigned nivolumab and 47 assigned chemotherapy with a follow-up of 6 months. Patients assigned nivolumab had an overall response rate of 32% compared with 11% for patients assigned chemotherapy. In addition, a reduction of at least 50% in the target lesion was seen in 82% of patients assigned nivolumab compared with 60% of patients assigned chemotherapy.

This study also evaluated nivolumab’s safety and found that the most common side effects were rash, itching, upper respiratory tract infections, and edema. Grade 3/4 adverse effects occurred in 9% of patients in the nivolumab arm compared with 31% of patients in the chemotherapy arm.

This accelerated approval of nivolumab came 3 months ahead of its scheduled review date of March 30, 2015.