SOUTH SAN FRANCISCO, Calif.—Onyx Pharmaceuticals Inc. recently announced thatthe U.S. Food and Drug Administration (FDA) has granted accelerated approval ofKyprolis (carfilzomib) for injection, a proteasome inhibitor that is indicated for thetreatment of patients with multiple myeloma who have received at least twoprior therapies, including bortezomib and an immunomodulatory agent, and havedemonstrated disease progression on or within 60 days of completion of the lasttherapy.
The indication for Kyprolis is based on response rate, the company reports, and currently, nodata are available for Kyprolis that demonstrate an improvement in progression-freesurvival or overall survival.
"Today's approval is a significant milestonefor Onyx and, most importantly, for patients with advanced myeloma who have fewtreatment options available to them," said Dr. N. Anthony Coles, president and CEO of Onyx Pharmaceuticals. "We deeplyappreciate the hundreds of patients who participated in the Kyprolis clinicalstudies that led to this accelerated approval, and recognize the manyclinicians across the country and researchers here at Onyx for their dedicationin bringing this promising new medicine to patients. We are committed tocontinuing the clinical development of Kyprolis across earlier stages ofmultiple myeloma treatment."
The approval was based on the results of the Phase IIb 003-A1 study, a single-arm, multicenter clinical trial that enrolled 266patients with multiple myeloma who had received a median of five prioranti-myeloma regimens. The primary efficacy endpoint was overall response, which turned out to be 22.9 percent, along with a median response duration of 7.8 months.
Safety data were evaluated in 526 patients withrelapsed and/or refractory multiple myeloma who received single-agentcarfilzomib, and the total deaths during the study numbered 37 deaths, or about 7 percent of patients. The mostcommon causes of death, other than disease progression, were cardiac (fivepatients), end-organ failure (four patients) and infection (four patients).
The most common serious adversereactions were pneumonia, acute renal failure, pyrexia and congestive heartfailure. The most common adversereactions (with an incidence of 30 percent or greater) observed in clinical trials of patientswith multiple myeloma were fatigue, anemia, nausea, thrombocytopenia, dyspnea,diarrhea and pyrexia. Serious adversereactions were reported in 45 percent of patients.
Still, Dr. David Siegel, chief of the Division of Multiple Myeloma at John Theurer CancerCenter at Hackensack University Medical Center, says, "This approval provides a new treatmentoption for the significant unmet need that exists in patients with multiplemyeloma who have progressed after use of available treatments. The single-agent activityof Kyprolis provides clinicians the opportunity to help these patients whountil now had no effective options."