The FDA granted accelerated approval to the injection drug carfilzomib for the treatment of patients with multiple myeloma who received at least two prior therapies, including bortezomib and an additional immunomodulatory agent.
Patients also must demonstrate disease progression on or within 60 days of the completion of the last therapy.
Carfilzomib (Kyprolis, Onyx Pharmaceuticals) is a proteasome inhibitor that disrupts the growth of cancer cells.
The FDA based its approval on the results of a phase 2, single-arm, multicenter trial in which researchers evaluated the efficacy of carfilzomib.
Researchers enrolled 266 patients with refractory multiple myeloma who received two prior lines of therapy, including bortezomib (Velcade, Millennium Pharmaceuticals) and thalidomide (Thalomid, Celgene) or lenalidomide (Revlimid, Celgene).
Investigators administered carfilzomib intravenously over 2 to 10 minutes on 2 consecutive days weekly for 3 weeks, followed by a 12-day rest period in a 28-day treatment cycle.
Patients received 20 mg/m² at each dose in cycle one and 27 mg/m² in ensuing cycles.
The overall response rate was 22.9% (95% CI, 18-25.5).
The most common adverse events observed in multiple clinical trials in patients with multiple myeloma were fatigue, anemia, nausea, thrombocytopenia, dyspnea, diarrhea and pyrexia.
Forty-five percent of patients reported serious adverse events, including pneumonia, acute renal failure, pyrexia and congestive heart failure.
Onyx will still submit a complete analysis of an ongoing randomized phase 3 trial comparing lenalidomide plus low-dose dexamethasone with lenalidomide plus low-dose dexamethasone plus carfilzomib as a condition of the accelerated approval.
Last month, the FDA’s Oncologic Drugs Advisory Committee expressed its support for carfilzomib’s approval, voting 11-0 with one abstention.