On July 20, 2012, the FDA granted accelerated approval to carfilzomib injection (Kyprolis; Onyx Pharmaceuticals) for the treatment of patients with multiple myeloma who have received at least 2 prior therapies, including bortezomib and an immunomodulatory agent, and who have demonstrated disease progression on or within 60 days of the completion of their last therapy.
The safety and effectiveness of carfilzomib was evaluated in a study of 266 patients participating in a single-arm, multicenter trial. Patients received carfilzomib intravenously for a 2- to 10-minute period on 2 consecutive days weekly for 3 weeks, followed by a 12-day rest period (a 28-day treatment cycle). Treatment was continued until disease progression, unacceptable toxicity, or completion of a maximum of 12 cycles.
The overall response rate was 22.9% and consisted of 1 complete response, 13 very good partial responses, and 47 partial responses. The median response duration was 7.8 months. The most common side effects observed in more than 30% of patients in the study were fatigue, low blood cell count and blood platelet levels, shortness of breath, diarrhea, and fever. Serious side effects included heart failure and shortness of breath.
As a condition of having received accelerated approval for carfilzomib, Onyx Pharmaceuticals will submit a complete analysis of an ongoing randomized phase 3 trial that compares lenalidomide plus low-dose dexamethasone to lenalidomide plus low-dose dexamethasone plus carfilzomib.