Onyx Pharma Receives FDA Fast-Track Approval For Carfilzomib (ONXX)

Onyx Pharma ONXX Receives FDA Fast-Track Approval For Carfilzomib. Onyx Pharmaceuticals, Inc. ONXX announced today that the U.S. Food and Drug Administration (FDA) has granted fast track designation for carfilzomib, a next generation proteasome inhibitor for the potential treatment of patients with relapsed and refractory multiple myeloma. The Fast Track designation process was developed by the FDA to facilitate the development, and expedite the review of drugs to treat serious or life-threatening diseases and address unmet medical needs. Positive complete results from the Phase 2b 003-A1 study evaluating single-agent carfilzomib in patients with relapsed and refractory multiple myeloma were recently announced at the American Society of Hematology (ASH) meeting in December 2010. Based on these results, Onyx has initiated a rolling submission of a New Drug Application (NDA) for potential accelerated approval of carfilzomib in the U.S. Through the Fast Track designation, Onyx is eligible to submit the carfilzomib NDA on a rolling basis, allowing Onyx to begin the NDA filing process immediately and giving the FDA an opportunity to review the completed sections of the registration application. Onyx has now submitted the non-clinical section of the carfilzomib NDA with the FDA and commenced its rolling NDA process. Onyx intends to complete its submission of the NDA for potential accelerated approval of carfilzomib in the U.S. as early as mid-2011. "The FDA's Fast Track designation recognizes the significant need for new treatment options for patients," said Ted Love, M.D., executive vice president, research and development and technical operations at Onyx. "We look forward to continuing to work with the FDA on the regulatory review and further development of carfilzomib, which has demonstrated encouraging response rates and tolerability in patients with advanced multiple myeloma. We are committed to making carfilzomib available to these very ill patients as quickly as possible." Carfilzomib was granted orphan drug designation by the FDA for the treatment of multiple myeloma in 2008. Orphan drug designation is granted to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases and disorders. Under the designation, the sponsor may be eligible for grant funding towards clinical trial costs, tax advantages, FDA user-fee benefits, and seven years of market exclusivity in the United States following drug approval by the FDA. About the Carfilzomib Development Program Carfilzomib is a selective, next generation proteasome inhibitor that has shown encouraging results in a broad clinical trial program in multiple myeloma. The carfilzomib development program includes a large, randomized international Phase 3 clinical trial, known as the ASPIRE trial, studying the combination of lenalidomide and low dose dexamethasone with or without carfilzomib in patients with relapsed multiple myeloma. The company has an agreement with the U.S. Food and Drug Administration (FDA) on a Special Protocol Assessment (SPA) and received Scientific Advice from the European Medicines Agency (EMA) on the design and planned analysis of the ASPIRE trial. A second Phase 3 clinical trial, called the FOCUS trial, is evaluating carfilzomib in patients with relapsed and refractory myeloma in Europe. Carfilzomib is also being evaluated in a broad investigator sponsored trial program including 1st line multiple myeloma, combination studies, lymphoma and other malignancies.