FDA Advisory Committee Votes in Favor of Pfizer’s MYLOTARG (gemtuzumab ozogamicin) for Acute Myeloid Leukemia


Pfizer Inc. (NYSE:PFE) today announced that the U.S. Food and Drug Administration’s (FDA) Oncologic Drug Advisory Committee (ODAC) voted that the results of ALFA-0701 demonstrated a favorable risk:benefit profile for MYLOTARG (gemtuzumab ozogamicin) 3 mg/m2 on days 1, 4 and 7 added to chemotherapy for patients with newly-diagnosed CD33-positive acute myeloid leukemia (AML). The role of the Advisory Committee is to provide recommendations to the FDA. The FDA decision on whether or not to approve the MYLOTARG application is anticipated by September 2017.

“We are extremely pleased with the Committee’s recommendation and believe this is an important step toward our goal of making MYLOTARG available to patients with newly-diagnosed AML,” said Mace Rothenberg, MD, Chief Development Officer, Oncology, Pfizer Global Product Development. “We look forward to working closely with the FDA as we continue the regulatory process. We are grateful to both the investigators who led MYLOTARG clinical trials and the patients who participated.”

The ODAC discussions were based on the Biologics License Application (BLA) currently under review by the FDA. The BLA includes Pfizer-sponsored studies from the original New Drug Application (NDA) for MYLOTARG, an investigator-led Phase 3 randomized, open-label study (ALFA-0701) and an individual patient data meta-analysis from over 3,000 patients in five randomized Phase 3 studies (including ALFA-0701).

These studies span 10 years of research and include more than 4,300 patients.“Clinical studies investigating MYLOTARG have provided a significant body of evidence supporting the risk:benefit profile of MYLOTARG in AML,” said Jorge Cortes, MD, University of Texas, MD Anderson Cancer Center. “Based on the totality of the efficacy and safety data, MYLOTARG, if approved, has the potential to be an important treatment option for adult patients with AML.”

Due to the critical unmet need for patients with newly-diagnosed AML, there has been great interest among AML investigators to evaluate MYLOTARG in this population using different doses and different schedules of MYLOTARG. These investigator-led clinical trials have provided more information on the efficacy and safety of MYLOTARG.

ODAC is an independent panel of experts that evaluates data concerning the efficacy and safety of marketed and investigational cancer treatments and makes recommendations to the FDA. Its vote is not binding, but is considered by the FDA in its decision-making process.

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