FDA panel backs carfilzomib

FDA's Oncologic Drugs Advisory Committee voted 11-0, with one abstention, that Kyprolis carfilzomib from Onyx Pharmaceuticals Inc. (NASDAQ:ONXX) has a favorable benefit-risk profile to treat patients with relapsed and refractory multiple myeloma who have received at least two prior lines of therapy that included a proteasome inhibitor and an immunomodulatory agent. Onyx is seeking accelerated approval for Kyprolis in the indication. The selective proteasome inhibitor has a July 27 PDUFA date.

The panel said Kyprolis' 22.9% overall response rate in the pivotal single-arm Phase II trial was indicative of clinical benefit. In briefing documents released ahead of the meeting, an FDA reviewer expressed concern about the cardiovascular toxicities and deaths seen in the trial. Ann Farrell, acting director of the Office of Hematology and Oncology Products, said at the meeting that the agency did not have "the same degree of concern." Moreover, most panel members said the level of serious adverse events was expected in patients with advanced disease who had received multiple treatments. The trial enrolled patients who had a median of five prior treatments. ...