FDA Expands Label for Carfilzomib in Multiple Myeloma

The FDA expanded the approval of carfilzomib for multiple myeloma

The US Food and Drug Administration (FDA) recently expanded the drug label for carfilzomib (Kyprolis) in patients with multiple myeloma. The drug is now approved in combination with dexamethasone or with lenalidomide plus dexamethasone for patients with relapsed or refractory disease who have received one to three lines of therapy. In addition, carfilzomib was also approved as a single agent for patients with myeloma who have relapsed on one or more prior therapies.

“Multiple myeloma remains an incurable disease where relapse inevitably occurs and over time patients become resistant to treatments,” Ruben Niesvizky, MD, director of the Multiple Myeloma Center at Weill Cornell Medicine and New York-Presbyterian/Weill Cornell Medical Center, said in a prepared statement released by the drug’s manufacturer Amgen. “As a clinician, I'm pleased with the tremendous progress that we have seen in the past 12 months in multiple myeloma treatment. This FDA approval is important because it provides physicians with flexible options for [carfilzomib] use in helping to manage this challenging disease.”

The expanded indication was granted based on results of the ENDEAVOR trial, published in Lancet Oncology in December. The trial randomly assigned 929 patients with relapsed or refractory myeloma to carfilzomib with dexamethasone or bortezomib with dexamethasone. Interim analysis results showed a median progression-free survival of 18.7 months in patients assigned carfilzomib compared with 9.4 months for patients assigned bortezomib (hazard ratio, 0.53 [95% CI, 0.44–0.65]; P < .0001).  The most frequent adverse events in patients assigned carfilzomib were anemia, diarrhea, dyspnea, fatigue, insomnia, pyrexia, and thrombocytopenia. Overall survival data are not yet mature.

In July, the FDA approved carfilzomib in combination with lenalidomide and dexamethasone for patients with myeloma who had received one to three prior lines of therapy. This approval was based on the results of the ASPIRE trial, a phase III trial that showed that carfilzomib prolonged progression-free survival compared with patients assigned to treatment with lenalidomide and dexamethasone alone (26.3 months vs 17.6 months).

Carfilzomib was first approved for mutliple myeloma in 2012, as a single agent in patients who had received at least two prior therapies including bortezomib and an immunomodulatory agent.