(By Balaseshan) Onyx Pharmaceuticals Inc. (NASDAQ:ONXX) shares touched a new 52-week high after the company received accelerated approval from the U.S. regulatory authorities for its blood cancer drug Kyprolis (carfilzomib).
The U.S. Food and Drug Administration (FDA) has granted approval of Kyprolis (carfilzomib) for Injection, 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.
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% 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% 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).
The most common serious adverse reactions were pneumonia, acute renal failure, pyrexia, and congestive heart failure. The most common adverse reactions (incidence of 30% 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% of patients.
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.
Multiple myeloma is a cancer of cells in the bone marrow that affects the production of red cells, white cells and stem cells and can damage bone. It is growing in numbers and affecting increasingly younger people.
Meanwhile, the International Myeloma Foundation (IMF) on April 27 said a new drug pomalidomide, developed by Celgene Corp. (NASDAQ:CELG), for multiple myeloma has been submitted to the FDA for approval. No new drugs have been approved for multiple myeloma in nearly six years. Pomalidomide will also be submitted for approval in Europe later this year.
ONXX is trading 12.94% higher at $77.25 on Friday. The stock has been trading between $27.17 and $79.20 for the past 52 weeks.