Onyx Pharmaceuticals, Inc., a California-based global biopharmaceutical company engaged in the development and commercialization of innovative therapies for improving the lives of people with cancer and other serious diseases, reported that the US Food and Drug Administration (FDA) has accepted the 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 FDA’s acceptance of the new drug application submission for carfilzomib marks an important milestone for bringing this promising therapy one step closer to patients with few remaining treatment options,” said Ted Love, MD, executive vice president, research and development and technical operations at Onyx Pharmaceuticals. “We believe that the efficacy and safety data make carfilzomib an appropriate candidate for accelerated approval in the relapsed and refractory treatment setting, and we look forward to hearing back from the FDA in the next few weeks regarding priority versus standard review.”

The submission is based on the 003-A1 study, an open-label, single-arm Phase 2b 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 percent 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.

Carfilzomib is being studied in several clinical trials either as a single-agent or in combination with other therapies, including: a phase III clinical trial, known as the ASPIRE trial, 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 company has an agreement with the FDA on a Special Protocol Assessment (SPA) on the design and planned analysis of the ASPIRE trial. A phase III clinical trial, called the FOCUS trial to support registration in Europe, is evaluating single-agent carfilzomib in patients with relapsed and refractory myeloma who have received three or more prior therapies. A phase II clinical trial, known as the 004 study, is evaluating single-agent carfilzomib in patients with relapsed and/or refractory multiple myeloma who have received one to three prior therapies. A phase I b/2 study, known as the 006 study, is evaluating carfilzomib in combination with lenalidomide and low dose dexamethasone in patients with relapsed and/or refractory myeloma.

A Multiple Myeloma Research Consortium (MMRC) phase I/II study is evaluating carfilzomib in combination with lenalidomide and low dose dexamethasone in newly diagnosed patients. This study is supported by Onyx Pharmaceuticals, MMRC, and Celgene Corporation. 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. An expanded access programme is underway in partnership with the Multiple Myeloma Research Foundation for eligible patients in the US, with relapsed and refractory multiple myeloma for whom no satisfactory treatment alternatives are available.

Multiple myeloma is the second most common haematologic cancer and results from an abnormality of plasma cells, usually in the bone marrow.

Onyx Pharmaceuticals, Inc. is a global biopharmaceutical company engaged in the development and commercialization of innovative therapies for improving the lives of people with cancer and other serious diseases.