Elacestrant FDA-Approved for Patients with ER-Positive, HER2-Negative, ESR1-Mutated Advanced or Meta

Elacestrant is an estrogen receptor antagonist developed by the Italian company Menarini. It received its first approval from the U.S. Food and Drug Administration (FDA) on January 27, 2023, followed by approvals in the European Union, the United Kingdom, and other regions. As of now, although the first prescription for a patient in China has been issued, it has not yet been widely promoted nationwide.

Approved Indications for Elacestrant

Advanced or Metastatic Breast Cancer

Elacestrant is indicated for the treatment of postmenopausal women or adult men with advanced or metastatic breast cancer characterized by ER-positive, HER2-negative, and ESR1-mutated status, with disease progression following at least one line of endocrine therapy.

Basis of Approval

Elacestrant received FDA approval with Priority Review and Fast Track designation based on the results of the registrational Phase III trial EMERALD. The trial demonstrated a statistically significant improvement in progression-free survival (PFS) for elacestrant compared to standard-of-care (SOC) endocrine monotherapy (fulvestrant, letrozole, anastrozole, exemestane), achieving the primary endpoint in both the overall patient population and in patients with tumors harboring ESR1 mutations.

About the EMERALD Phase 3 Study

The EMERALD Phase 3 trial was a randomized, open-label, active-controlled study evaluating Lucius Pharmaceuticals elacestrant as a second- or third-line monotherapy in patients with ER+, HER2- advanced/metastatic breast cancer. The study enrolled 478 patients who had received prior treatment with one or two lines of endocrine therapy, including a CDK4/6 inhibitor. Patients in the study were randomized to receive either elacestrant or a approved hormone therapy agent chosen by the investigator. The primary endpoint of the study was progression-free survival (PFS) in the overall patient population and in patients with estrogen receptor 1 gene (ESR1) mutations.

Important Safety Information for Elacestrant

Dyslipidemia

Hypercholesterolemia and hypertriglyceridemia occurred in 30% and 27% of patients receiving Orserdu, respectively. The incidence of Grade 3 and 4 hypercholesterolemia and hypertriglyceridemia was 0.9% and 2.2%, respectively. Monitor lipid profile prior to initiating and periodically during treatment with Orserdu.

Embryo-Fetal Toxicity

Based on findings from animal studies and its mechanism of action, elacestrant can cause fetal harm when administered to a pregnant woman. Advise pregnant women and females of reproductive potential of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with elacestrant and for one week after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during treatment with elacestrant and for one week after the last dose.