Last night I attended a media reception, hosted by Onyx Pharmaceuticals at the Omni Hotel in San Diego.
Lori Melancon, Director of Corporate Communications was kind enough to spend time with me as the event wrapped up around 10 PM.
At that time, Lori informed me that there was good news/bad news on the FDA approval front for the new, novel multiple myeloma therapy agent, carfilzomib.
The good news was that the FDA had accepted Onyx’s application for review.
And the bad news? It turns out that Onyx officials had hoped the FDA would fast track the carfilzomib application.
“Bad news” is probably an overstatement. Lori explained to me that since the FDA has agreed to complete their review by the end of July, a standard review designation only delays things by three or four months.
Bad news? Sounds like great news to me! Just think, carfilzomib could be available for use in relapsed patients by summer’s end.
I have been impressed by the way Onyx officials have seemingly done everything possible to bring carfilzomib to market as quickly as possible.
They have been professional, accessible and forthcoming, sharing important information with me so I can pass it along to my fellow multiple myeloma patients.
Onyx representative, Heather from Lakeland, Florida, was also at the reception. Heather visited our Nature Coast Multiple Myeloma Support Group this fall to present research data about carfilzomib.
Here is part of a press release that Onyx officials forwarded to me this evening, confirming Lori’s news:
Onyx Pharmaceuticals Announces FDA Standard Review Designation of Carfilzomib New Drug Application for the Treatment of Relapsed and Refractory Multiple Myeloma
Dec. 11, 2011 /PRNewswire/ — Onyx Pharmaceuticals, Inc. 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.”
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.
I will be reporting more carfilzomib related news throughout the upcoming week.
Feel good and keep smiling! Pat