FDA Greenlights Lilly Early Breast Cancer Drug - with a Caveat

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Eli Lilly announced that the U.S. Food and Drug Administration (FDA) had approved its Verzenio (abemaciclib) for a specific type of early breast cancer that is at high risk of recurrence after surgery. 

The drug is limited to patients who test at least 20% for a Ki-67 score on an FDA-approved test. Ki-67 is a marker of cellular proliferation. This limitation is likely to make the rollout of the drug to be a bit tougher.

The specific approval is for Verzenio in combination with endocrine therapy, such as tamoxifen or an aromatase inhibitor, for adjuvant treatment of adults with hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-), node-positive, early breast cancer (EBC) at high risk of recurrence and a Ki-67 score of greater than or equal to 20%. Verzenio is a CDK4/6 inhibitor.

“Over time, the collective results of the Verzenio clinical development program have demonstrated a differentiated CDK4/6 inhibitor profile, and the landmark data from the monarchE trial that supported this new indication in HR+ HER2- early breast cancer represent another important step forward for people who are in need of new treatment options,” said Jacob Van Naarden, Senior Vice President, Chief Executive Officer of Loxo Oncology at Lilly and President, Lilly Oncology. “We are pleased with this initial approval in the adjuvant setting and as these data continue to mature, we look forward to further opportunities to work with health authorities to expand the use of Verzenio in this setting.”

This is the first CDK4/6 inhibitor approved for this setting. In 2020, Pfizer's Phase III trial of its own CDK4/6 inhibitor, Ibrance, failed to hit its primary endpoint in this setting. Novartis is evaluating its Kisqali for this indication but won’t have data available until late 2022.

The Lilly approval was based on the Phase III monarchE study. Patients were randomized to receive two years of Verzenio twice a day plus the physician’s choice of standard endocrine therapy or standard endocrine therapy alone. They continued to receive adjuvant endocrine therapy for up to five to 10 years. The primary endpoint was invasive disease-free survival (IDFS), which was met at a pre-specific interim analysis. It demonstrated statistically significant improvement in IDFS for patients receiving Verzenio plus ET compared to the patients receiving ET alone.

A pre-specified analysis was also run of IDFS in patients with high-risk clinical and pathological factors and a Ki-67 score of 20% or greater. This included patients with five or higher positive axillary lymph nodes (ALN) or 1-3 positive ALN with either Grade 3 disease and/or tumor size of 5 cm or greater and whose tumors had a Ki-67 score of 20% or greater. This group also had a statistically significant improvement in IDFS.

“Women and men living with high-risk HR+ HER2- early breast cancer want to do all they can to reduce the risk of the disease coming back, with the hope of living free of cancer,” said Jean Sachs, Chief Executive Officer, Living Beyond Breast Cancer. “This approval of Verzenio provides a new treatment option to help them do just that. This approval brings new optimism to the breast cancer community.”

The company expects to present data supporting the approval at the European Society for Medical Oncology (ESMO) Virtual Plenary on October 14.