For immediate release:

Today, the U.S. Food and Drug Administration has approved Enhertu (fam-trastuzumab-deruxtecan-nxki), an intravenous infusion for the treatment of patients with HER2-low inoperable (cannot be removed) or metastatic (spread to other parts of the body). breast cancer. This is the first approved therapy targeting patients with the HER2-low breast cancer subtype, which is a new subset of HER2-negative breast cancer.

An estimated 287,850 new cases of female breast cancer will be diagnosed in 2022 in the United States. About 80-85% of these new cases were previously considered an HER2-negative subtype (including hormone receptor-positive and triple-negative breast cancer), which means that the tumors do not overexpress or make too many copies of the HER2 protein. Of that percentage of breast cancer diagnoses, approximately 60% of patients previously classified as having the HER2-negative subtype can now be considered as HER2-low. Prior to today’s approval, patients with low HER2 received either endocrine therapy or chemotherapy.

“Today’s approval highlights the FDA’s commitment to being at the forefront of scientific advancements by making targeted cancer treatment options available to more patients,” said Richard Pazdur, MD, director of the Cancer Center of Excellence. FDA and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research. “Having therapies specifically tailored to each patient’s cancer subtype is a priority to ensure access to safe and innovative treatments.”

As part of the administration’s Cancer Moonshot program, President Biden leveraged federal agencies to develop ways to reduce the rate of cancer deaths and improve the lives of cancer patients and their families through advances in research and technology. on cancer and the development of new programs. Enhertu’s approval further illustrates how the FDA’s efforts align with Cancer Moonshot’s goals of targeting the right treatments to the right patients, accelerating progress against the deadliest and rarest cancers, and learning from the experience of all patients.

HER2 receptors, which are proteins produced by the HER2 gene, are important in determining a patient’s treatment. HER2-negative includes hormone receptor positive and triple negative breast cancers. HER2-low is a new classification of the HER2 subtype. It describes a new breast cancer subtype that has some HER2 proteins on the cell surface, but not enough to be classified as HER2-positive.

Patients with HER2-low breast cancer are eligible for Enhertu if they have received prior metastatic chemotherapy or if the cancer has returned during or within 6 months of completing adjuvant chemotherapy.

This approval is based on DESTINY-Breast04, a randomized, multicenter, open-label clinical trial that enrolled 557 adult patients with unresectable or metastatic HER2-low breast cancer. The study included two cohorts: 494 patients with hormone receptor positive (HR +) and 63 patients with hormone receptor negative (HR-). Of these patients, 373 randomly received Enhertu by intravenous infusion every three weeks and 184 randomly received physician-chosen chemotherapy (eribulin, capecitabine, gemcitabine, nab paclitaxel, or paclitaxel). The results showed improved both progression-free survival and overall survival in people with unresectable or metastatic HER2-low breast cancer.

The mean age of the study participants was 57 years, ranging in age from 28 to 81 years. Among the 557 patients, 24% were 65 or older. Females made up 99.6% of the test population. The race of trial participants was reported as 48% White, 40% Asian, 2% Black or African American, and 3.8% Hispanic / Latino.

The most common adverse reactions in patients who received Enhertu in DESTINY-Breast04 are nausea, fatigue, alopecia, vomiting, constipation, decreased appetite, musculoskeletal pain and diarrhea. The prescribing information includes a boxed warning to alert healthcare professionals of the risk of interstitial lung disease and embryo-fetal toxicity. Enhertu is not recommended for pregnant women.

Enhertu received priority review and breakthrough therapy designations for this indication. The FDA granted Enhertu approval to Daiichi Sankyo four months before the Prescription Drug User Fee Act (PDUFA) expired.

This review was conducted as part of the Orbis Project, an initiative of the FDA’s Cancer Center of Excellence. The Orbis project provides a framework for the simultaneous submission and review of cancer drugs among international partners. For this review, the FDA partnered with the Australian Therapeutic Goods Administration, Health Canada and Swissmedic in Switzerland. Application reviews may be ongoing at other regulatory agencies.

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The FDA, an agency within the U.S. Department of Health and Human Services, protects public health by ensuring the safety, efficacy, and protection of drugs, vaccines, and other biological products for human use and medical devices for human and veterinary use. The agency is also responsible for our nation’s food safety and supply, cosmetics, dietary supplements, products that emit electronic radiation, and for regulating tobacco products.