FDA approves Onyx Pharmaceuticals' Kyprolis for multiple myeloma
SOUTH SAN FRANCISCO, Calif.óOnyx Pharmaceuticals Inc. recently announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval of Kyprolis (carfilzomib) for injection, a proteasome inhibitor that is indicated for the treatment of patients with multiple myeloma who have received at least two prior therapies, including bortezomib and an immunomodulatory agent, and have demonstrated disease progression on or within 60 days of completion of the last therapy.
The indication for Kyprolis is based on response rate, the company reports, and currently, no data are available for Kyprolis that demonstrate an improvement in progression-free survival or overall survival.
"Today's approval is a significant milestone for Onyx and, most importantly, for patients with advanced myeloma who have few treatment options available to them," said Dr. N. Anthony Coles, president and CEO of Onyx Pharmaceuticals. "We deeply appreciate the hundreds of patients who participated in the Kyprolis clinical studies that led to this accelerated approval, and recognize the many clinicians across the country and researchers here at Onyx for their dedication in bringing this promising new medicine to patients. We are committed to continuing the clinical development of Kyprolis across earlier stages of multiple myeloma treatment."
The approval was based on the results of the Phase IIb 003-A1 study, a single-arm, multicenter clinical trial that enrolled 266 patients with multiple myeloma who had received a median of five prior anti-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 with relapsed and/or refractory multiple myeloma who received single-agent carfilzomib, and the total deaths during the study numbered 37 deaths, or about 7 percent of patients. The most common causes of death, other than disease progression, were cardiac (five patients), end-organ failure (four patients) and infection (four patients).
The most common serious adverse reactions were pneumonia, acute renal failure, pyrexia and congestive heart failure. The most common adverse reactions (with an incidence of 30 percent or greater) observed in clinical trials of patients with multiple myeloma were fatigue, anemia, nausea, thrombocytopenia, dyspnea, diarrhea and pyrexia. Serious adverse reactions were reported in 45 percent of patients.
Still, Dr. David Siegel, chief of the Division of Multiple Myeloma at John Theurer Cancer Center at Hackensack University Medical Center, says, "This approval provides a new treatment option for the significant unmet need that exists in patients with multiple myeloma who have progressed after use of available treatments. The single-agent activity of Kyprolis provides clinicians the opportunity to help these patients who until now had no effective options."