FDA had flagged life-threatening safety issues that could have delayed approval of Onyx’s myeloma drug Kyprolis. But in the end, the agency instead opted for explicit instructions in the label to avoid risks, stopping short of a boxed warning, and a sub-study in a Phase III trial to fully assess cardiac and pulmonary risks.

Onyx is pricing new myeloma drug carfilzomib at $9,950 per cycle, 65% higher than Takeda/Millennium’s pioneering proteasome inhibitor Velcade. Launch plans include financial assistance for providers, comprehensive patient assistance program and transition for expanded access patients to commercial drug.

FDA’s Oncologic Drugs Advisory Committee overwhelmingly endorsed the second-generation proteasome inhibitor for accelerated approval in the relapsed/refractory population. Onyx’s reliance on a single-arm study was appropriate given the lack of treatment options in a heavily pre-treated population, said ODAC members, who also looked favorably upon the company’s advanced plans for confirmatory trials.