Onyx Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval of Kyprolis (carfilzomib) for Injection.
Kyprolis (carfilzomib), a proteasome inhibitor, 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. 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 N. Anthony Coles, M.D., President and Chief Executive Officer 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."
According to a release, the approval was based on the results of the Phase 2b 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 (ORR) and determined by an Independent Review Committee using the International Myeloma Working Group (IMWG) criteria. ORR was 22.9 percent and median response duration was 7.8 months.
Safety data were evaluated in 526 patients with relapsed and/or refractory multiple myeloma who received single-agent carfilzomib. There were 37 deaths on study, or 7 percent of patients. The most common causes of death, other than disease progression, were cardiac (5 patients), end-organ failure (4 patients), and infection (4 patients). 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. The most common serious adverse reactions were pneumonia, acute renal failure, pyrexia, and congestive heart failure. The most common adverse reactions (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.
"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," said Dr. David Siegel, Chief of the Division of Multiple Myeloma at John Theurer Cancer Center at Hackensack University Medical Center. "The single-agent activity of Kyprolis provides clinicians the opportunity to help these patients who until now had no effective options."
Enrollment has been completed for the Phase 3 confirmatory clinical trial, known as the ASPIRE trial. The company has an agreement with the FDA on a Special Protocol Assessment (SPA) for this trial.
Onyx also announced the availability of Onyx Pharmaceuticals 360, a patient and caregiver support and services program, designed to help patients navigate the treatment journey, including reimbursement and payment support, treatment support and referrals to third-party organizations for day-to-day and emotional support.
Multiple myeloma is the second most common hematologic cancer and results from an abnormality of plasma cells, usually in the bone marrow. In the United States, more than 50,000 people are living with multiple myeloma and approximately 20,000 new cases are diagnosed annually. Worldwide, more than 220,000 people are living with multiple myeloma and approximately 100,000 new cases are diagnosed annually.
Kyprolis is approved by the U.S. Food and Drug Administration (FDA) 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. Approval is based on response rate. Clinical benefit, such as improvement in survival or symptoms, has not been verified.
Kyprolis is being studied in several clinical trials either as a single-agent or in combination with other therapies, including:
-A global Phase 3 clinical trial, known as the ASPIRE trial, has completed enrollment and is evaluating the combination of lenalidomide and low-dose dexamethasone with or without Kyprolis in patients with relapsed multiple myeloma who have received one to three prior therapies. The company has an agreement with the FDA on a Special Protocol Assessment (SPA) and has received Scientific Advice from the European Medicines Agency (EMA) on the design and planned analysis of the trial.
-A Phase 3 clinical trial, known as the FOCUS trial, is evaluating single-agent Kyprolis in patients with relapsed and refractory myeloma who have received three or more prior therapies. The trial is designed to facilitate regulatory approvals around the world.
-A global head-to-head Phase 3 clinical trial, known as the ENDEAVOR trial, is evaluating the combination of Kyprolis and low- dose dexamethasone versus the combination of bortezomib and low- dose dexamethasone.
-A Phase 1/2 study being conducted by Onyx's partner Ono Pharmaceutical Co. is evaluating Kyprolis in Japanese patients with relapsed/refractory multiple myeloma.
Onyx Pharmaceuticals, Inc. is a global biopharmaceutical company engaged in the development and commercialization of therapies for improving the lives of people with cancer.
More information:
http://www.onyx.com
Source: Pharmacychoice
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