Accelerated approval for pertuzumab in the neoadjuvant setting: winds of change?

Accelerated approval for agents that improve the frequency of complete pathologic response in the primary breast cancer setting heralds a broadening of the opportunities to get effective agents to the market more quickly. However, these new pathways will require identifying the signature or subtype for which the agent is most effective, and evidence of enrollment of patients to a trial that enables the ascertainment of event-free survival. The recent approval of pertuzumab for use in the neoadjuvant setting is evidence that the FDA is committed to supporting the accelerated approval pathway. The situations in which approval is likely to be granted are discussed. ©2014 American Association for Cancer Research.