The FDA has accepted Onyx Pharmaceuticals' New Drug Application (NDA)
submission for carfilzomib, a next generation proteasome inhibitor, for the potential treatment of patients with relapsed and refractory multiple myeloma.
The submission is based on the 003-A1 study, an open-label, single-arm phase IIb trial. The trial evaluated 266 heavily-pretreated patients with relapsed and refractory multiple myeloma who had received at least two prior therapies, including bortezomib and either thalidomide or lenalidomide. Refractory disease was defined as less than or equal to a 25% response or progression during therapy, or progression within 60 days after completion of therapy. The primary endpoint was overall response rate. Secondary endpoints included duration of response, clinical benefit rate, overall survival, time-to-progression, progression-free survival, and safety. Safety data from additional carfilzomib studies were also included in the submission.