Onyx Pharmaceuticals recently announced that the FDA’s Oncologic Drugs Advisory Committee (ODAC) determined by a vote of 11–0 (with 1 abstention) that, in patients with relapsed and refractory multiple myeloma who have received at least two prior lines of therapy that included a proteasome inhibitor and an immunomodulatory agent, the benefit-risk assessment is favorable for the use of carfilzomib (proposed brand name, Kyprolis).
Onyx is developing carfilzomib for use in multiple myeloma across a variety of treatment lines. The Prescription Drug User Fee Act (PDUFA) date for completion of FDA review of the carfilzomib New Drug Application (NDA) for accelerated approval is July 27, 2012.
About Carfilzomib
The carfilzomib NDA submission is based on the 003-A1 study, an open-label, single-arm phase IIb trial as well as supportive data from additional studies. The 003-A1 trial evaluated 266 heavily pretreated patients with relapsed and refractory multiple myeloma who had received at least two prior therapies, including bortezomib (Velcade) and either thalidomide (Thalomid) or lenalidomide (Revlimid).
Carfilzomib is being studied in several clinical trials either as a single-agent or in combination with other therapies, including:
- A global phase III clinical trial, known as the ASPIRE trial, has completed enrollment and is evaluating the combination of lenalidomide and low-dose dexamethasone with or without carfilzomib in patients with relapsed multiple myeloma who have received one to three prior therapies.
- The phase III FOCUS trial is evaluating single-agent carfilzomib 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 phase III clinical trial, called ENDEAVOR, is planned to begin enrolling patients in mid-2012. This head-to-head trial will evaluate the combination of carfilzomib and low-dose dexamethasone vs the combination of bortezomib and low-dose dexamethasone.
- A phase I/II study being conducted by Onyx’s partner Ono Pharmaceutical Co, Ltd, is evaluating carfilzomib in Japanese patients with relapsed/refractory multiple myeloma. ■
