FDA Grants Accelerated Approval for Carfilzomib in Relapsed/Refractory Multiple Myeloma
This indication is based on an overall response rate of 22.9%, determined by an Independent Review Committee using the International Myeloma Working Group (IMWG) criteria, observed in a phase 2B, single-arm, multicenter trial that enrolled 266 patients with multiple myeloma who had received a median of 5 prior anti-myeloma regimens, according to Onyx Pharmaceuticals, the manufacturer of carfilzomib. Median response duration was 7.8 months.
Of the 526 patients who received single-agent carfilzomib in which safety was evaluated, 37 (7%) died; other than disease progression, deaths included cardiac (n=5), end-organ failure (n=4), and infection (n=4).
“Important warnings and precautions include cardiac arrest, congestive heart failure, myocardial ischemia; pulmonary hypertension, pulmonary complications, infusion reactions, infusion reactions, tumor lysis syndrome, thrombocytopenia, hepatic toxicity and embryo-fetal toxicity,” Onyx noted. Serious adverse reactions, reported in 45% of patients, were pneumonia, acute renal failure, pyrexia, and congestive heart failure. Adverse reactions ≥30% were fatigue, anemia, nausea, thrombocytopenia, dyspnea, diarrhea, and pyrexia.
The phase 3 global confirmatory clinical trial, ASPIRE—evaluating lenalidomide and low-dose dexamethasone with or without carfilzomib in patients with relapsed multiple myeloma who have received 1 to 3 prior therapies—has completed enrollment.
Other phase 3 trials include FOCUS, in which carfilzomib is being investigated as a single agent in patients with relapsed and refractory myeloma, and ENDEAVOR, which is evaluating carfilzomib plus low-dose dexamethasone vs bortezomib plus low-dose dexamethasone.
Currently, no data are available that demonstrate improved progression-free survival or overall survival for carfilzomib, a proteasome inhibitor.
Onyx is also launching a comprehensive patient and caregiver support and services program, Onyx 360.
Link to press release:
Link to prescribing information: