FDA Grants Accelerated Approval for Carfilzomib in Relapsed/Refractory Multiple Myeloma

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(ChemotherapyAdvisor) – The U.S. Food and Drug Administration granted accelerated approval of carfilzomib (Kyprolis™) for injection for the treatment of patients with multiple myeloma who have received at least two prior therapies, including bortezomib and an immunomodulatory agent, with disease progression on or within 60 days of previous therapy.

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.

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