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In Historic Vote, ODAC Suggests Approval of Neoadjuvant Pertuzumab Regimen

Ben Leach
Published: Thursday, Sep 12, 2013

Dr Mikkael Sekeres

Mikkael Sekeres, MD, MS

The FDA’s Oncologic Drugs Advisory Committee (ODAC) voted 13-0 with one abstention in support of pertuzumab (Perjeta) in combination with trastuzumab and docetaxel for patients with HER2-positive breast cancer in the neoadjuvant setting. If the FDA follows the recommendation of ODAC, this pertuzumab regimen would be the first neoadjuvant regimen formally approved by the FDA for any type of cancer.

The favorable reaction by ODAC bodes well for the eventual accelerated approval of pertuzumab in this setting. While the FDA is not required to follow the recommendations of ODAC, historically, the federal agency often does. The FDA is scheduled to make a decision regarding the approval of the pertuzumab regimen in this setting on or before October 31, 2013.

Today’s ODAC meeting was the first in which the committee was asked to consider recommending an oncologic drug for approval on the basis of pathologic complete response (pCR) as the primary endpoint in the pivotal clinical trials. The committee was asked to consider the question of whether pertuzumab demonstrated “a favorable benefit to risk evaluation for the neoadjuvant treatment of early breast cancer.”

“It’s not every day that this committee gets to consider a new indication in breast cancer,” said Mikkael Sekeres, MD, MS, associate professor of Medicine in the department of Hematologic Oncology and Blood Disorders at the Cleveland Clinic Taussig Cancer Institute in Ohio and ODAC chairperson.

If this pertuzumab regimen receives FDA approval, the decision could be precedent-setting and result in much earlier approvals for drugs in earlier disease settings.

Dr. Jose Baselga

José Baselga, MD, PhD

“Early disease is the setting where we can have the biggest impact on long-term survival,” said Jose Baselga, MD, physician-in-chief at Memorial Sloan-Kettering Cancer Center in New York, in a presentation before ODAC today. Baselga served as primary investigator of the CLEOPATRA trial, which resulted in the FDA’s approval of this same pertuzumab regimen in the metastatic setting, and he is also a primary investigator in the APHINITY trial, which is evaluating the efficacy of this pertuzumab regimen in the adjuvant setting.

Baselga noted that, typically, the time between a drug’s approval in the metastatic setting and its subsequent approval in an earlier setting is “simply much too long.” He gave the examples of docetaxel and trastuzumab, where eight years passed between the approvals of each drug in the metastatic and adjuvant settings, respectively. In the case of pertuzumab, Baselga said that its efficacy has been well established in clinical trials and will continue to be assessed in the APHINITY trial in the adjuvant setting, with results expected to become available in late 2016.

However, Baselga indicated that an approval of pertuzumab in the neoadjuvant setting now, a full three years prior to when those results will become available, could have an immediate impact on patients.

“In terms of survival, even though trastuzumab has had a profound impact, a substantial number of patients are still dying from HER2-positive disease,” Baselga said. “I feel strongly that we should make this therapy available to [patients] now.”

Much of the discussion among members of ODAC concerned the potential precedents set by approving an oncologic drug in the neoadjuvant setting. The pivotal trials at the center of this supplemental biologics license application were relatively small compared with many of the clinical trials that have resulted in the approval of drugs for breast cancer in the metastatic setting, enrolling on the order of a couple of hundred patients as opposed to multiple thousands of patients. Therefore, these trials were not powered to determine survival information. Despite several meta-analyses showing an association between pCR and survival benefits, no definitive answer has been derived from these studies as of yet.




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