I received this disappointing, although not unexpected e-mail from Lori Murray, Director, Corporate Communications & Investor Relations for Onyx Pharmaceuticals late yesterday:
I wanted to make you aware of the announcement we issued this afternoon since you interact with so many people with myeloma.
We announced that we are delaying the New Drug Application (NDA) filing for carfilzomib based on a recent meeting with the Chemistry, Manufacturing & Controls (CMC) review division of the U.S. Food & Drug Administration (FDA). The CMC is the part of the FDA that regulates the physical drug substance and product, including manufacturing.
Importantly, we have had productive interactions with the FDA, including a pre-NDA meeting with the clinical division. We remain confident in the clinical and preclinical data package supporting the NDA and in our manufacturing strategy. The clinical profile of carfilzomib has not changed and no new safety signals have been observed. Our Phase 3 program is on track and actively enrolling patients, and this delay is not expected to impact any of our carfilzomib trials or other development plans.
In preparation for commercial-scale production, we made process enhancements in the production of carfilzomib. As a result of those enhancements, the FDA has requested additional CMC information to assess the impact of these changes on commercial-scale manufacturing. Process enhancements are customary within the industry when transitioning from clinical to commercial-scale manufacturing.
Specifically, in a recently completed manufacturing-scale run, we observed minor variations in the process that we believe are primarily related to equipment temperature variations. In response to the FDA’s request, we are providing the agency with additional CMC information on these variations to support our process enhancements. We already have additional data in hand and are generating more to share as part of our ongoing dialogue with the CMC division in preparation for an NDA filing. We believe that by providing this supportive data, we will strengthen our NDA package for filing and approval of carfilzomib. We now expect to file the NDA as early as mid-year 2011 for potential accelerated approval.
Our excitement about the potential of carfilzomib and our commitment to bringing this important potential therapy to patients with multiple myeloma remains unchanged. We would like to underscore that this is a timing issue; we believe the opportunity for carfilzomib is significant, and we will be working with the FDA to make carfilzomib available to patients as quickly as possible.
Thank you for your continued commitment to patients who follow you on your blog and via Myeloma Beacon.
I would like to thank Lori for taking the time to forward us this detailed explanation of Onyx’s decision to delay the carfilzomib application. I’m sure we will be hearing much more about this over the coming weeks. I will work to help keep all of us informed.
Feel good and keep smiling! Pat