-- Conference Call Scheduled for Monday, December 12, 2011, at 5:00 a.m. PT --
SOUTH SAN FRANCISCO, Calif., Dec. 11, 2011 /PRNewswire/ -- Onyx Pharmaceuticals, Inc. (Nasdaq: ONXX) announced today that the U.S. Food and Drug Administration (FDA) has granted Standard Review designation to the New Drug Application (NDA) for carfilzomib for the potential treatment of patients with relapsed and refractory multiple myeloma. The Prescription Drug User Fee Act (PDUFA) date for completion of review by the FDA of the NDA is July 27, 2012.
In the NDA filing designation letter from the FDA, received December 9th, the agency advised the company that recent Oncology Drug Advisory Committee recommendations specify a preference for Phase 3 trials for the accelerated approval pathway. Based on its preliminary review and ongoing assessment of the application, the FDA outlined potential review issues including whether the application is sufficient to support an FDA conclusion that the data provided within the NDA meets accelerated approval criteria and whether the benefit and risk are appropriately balanced, given that the application is based on a single-arm study.
"Our goal remains to make this promising new therapy available to patients with relapsed and refractory multiple myeloma as quickly as possible," said Ted W. Love, M.D., executive vice president, research and development and technical operations at Onyx Pharmaceuticals. "We are committed to working with the agency to address the comments outlined in the letter."
Investor Teleconference
The Onyx management team will host a teleconference at 5:00 a.m. PT on December 12, 2011, to discuss the NDA review designation. Interested parties may access the teleconference and the presentation that accompanies it on our website at:
http://www.onyx-pharm.com/investors/event-calendar
or by dialing 1-847-585-4405 and using the passcode 31404173. A replay of the presentation will be available on the Onyx website or by dialing 1-630-652-3042 and using the passcode 31404173# later in the day. The replay will be available on the Onyx website until December 26, 2011.
About the Phase 2b 003-A1 Study
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.(i) 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. The safety database in the NDA includes 526 patients with multiple myeloma.
Carfilzomib Development Program
Carfilzomib is being studied in several clinical trials either as a single-agent or in combination with other therapies, including:
- A Phase 3 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 3 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 1/2 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 program is underway in partnership with the Multiple Myeloma Research Foundation for eligible patients in the U.S., with relapsed and refractory multiple myeloma for whom no satisfactory treatment alternatives are available.
About Multiple Myeloma
Multiple myeloma is the second most common hematologic cancer and results from an abnormality of plasma cells, usually in the bone marrow. In the United States, more than 50,000 people are living with multiple myeloma and approximately 20,000 new cases are diagnosed annually.(ii) Worldwide, more than 180,000 people are living with multiple myeloma and approximately 86,000 new cases are diagnosed annually.(iii)
About Onyx Pharmaceuticals, Inc.
Based in South San Francisco, California, 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. The company is focused on developing novel medicines that target key molecular pathways. For more information about Onyx, visit the company's website at www.onyx-pharm.com.
Forward Looking Statements
This news release contains "forward-looking statements" of Onyx within the meaning of the federal securities laws. These forward-looking statements include without limitation, statements regarding the progress and results of the clinical development, the expanded access program, safety, regulatory processes, commercialization efforts or commercial potential of carfilzomib. These statements are subject to risks and uncertainties that could cause actual results and events to differ materially from those anticipated, including risks related to the development and commercialization of pharmaceutical products. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Reference should be made to Onyx's Annual Report on Form 10-K for the year ended December 31, 2010, filed with the Securities and Exchange Commission under the heading "Risk Factors" and Onyx's Quarterly Reports on Form 10-Q for a more detailed description of such factors. Readers are cautioned not to place undue reliance on these forward-looking statements that speak only as of the date of this release. Onyx undertakes no obligation to update publicly any forward-looking statements to reflect new information, events, or circumstances after the date of this release except as required by law.
(i) http://www.fda.gov/forconsumers/byaudience/forpatientadvocates/speedingaccesstoimportantnewtherapies/ucm128291.htm
(ii) National Cancer Institute, Surveillance Epidemiology and End Results, 2007 Facts and Figures
(iii) International Agency for Research on Cancer, GLOBOCAN 2002 database
SOURCE Onyx Pharmaceuticals, Inc.