Menarini Group's ORSERDU® (Elacestrant) Receives Positive CHMP Opinion for European Commission Approval in Locally Advanced or Metastatic Breast Cancer with ESR1 Activating Mutation
Saturday, July 22, 2023
The Menarini Group, a renowned international pharmaceutical and diagnostics company, along with its subsidiary Stemline Therapeutics Inc., has received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) for the approval of ORSERDU® (elacestrant) as a monotherapy. This approval is intended for the treatment of postmenopausal women and men with locally advanced or metastatic breast cancer that is estrogen receptor (ER) positive, HER2 negative, and who carry an activating mutation in the ESR1 gene, and have experienced disease progression after at least one line of endocrine therapy, including a CDK 4/6 inhibitor.
The CHMP's opinion will now be reviewed by the European Commission, which has the authority to grant marketing authorization for human medicines throughout the European Union (EU). If approved, ORSERDU will be marketed by Stemline and its affiliates in Europe. This therapy will be the first and only treatment specifically indicated for ER+, HER2- tumors with mutations in the ESR1 gene. ESR1 mutations are acquired mutations that occur due to exposure to endocrine therapy and are found in up to 40% of patients with ER+, HER2- metastatic breast cancer. These mutations are known to be a factor in resistance to standard endocrine therapy, making such tumors more challenging to treat.
Elcin Barker Ergun, CEO of the Menarini Group, expressed that ORSERDU's potential approval represents a significant step forward in addressing the unmet needs of patients living with metastatic breast cancer. It would offer an important new treatment option for those affected by ESR1, ER+, HER2- metastatic breast cancer, which currently lacks specific therapies.
The positive CHMP opinion for ORSERDU is based on data from the Phase 3 clinical trial EMERALD, which demonstrated a statistically significant improvement in progression-free survival (PFS) with elacestrant compared to standard of care (SOC), defined as the investigator's choice of an approved endocrine monotherapy. Specifically, in patients with ESR1 mutations, elacestrant achieved a median PFS of 3.8 months compared to 1.9 months with standard care, leading to a 45% reduction in the risk of progression or death.
Furthermore, a post-hoc subgroup analysis of the EMERALD trial revealed that the duration of prior treatment with CDK4/6 inhibitors was positively associated with longer PFS with elacestrant. Patients with mutations in the ESR1 gene who were treated with CDK4/6 inhibitors for ≥12 months prior to randomization in the study experienced a median PFS of 8.6 months with elacestrant compared to 1.9 months with standard care, resulting in a 59% reduction in the risk of progression or death.Giuseppe Curigliano, MD, PhD, Professor of Medical Oncology at the University of Milan, praised the potential of elacestrant as a remarkable treatment option for patients with advanced or metastatic breast cancer who have ER+, HER2-, and ESR1 gene mutations.
The safety data of ORSERDU were consistent with previously reported results. The most common adverse reactions were manageable and included nausea, increased triglycerides, increased cholesterol, vomiting, fatigue, dyspepsia, diarrhea, and more.
Elacestrant is also being investigated in other clinical studies in metastatic breast cancer, either alone or in combination with other therapies, and there are plans to evaluate it in early-stage breast cancer.
In summary, the positive CHMP opinion for ORSERDU represents a promising advancement in the treatment of metastatic breast cancer with specific genetic mutations, potentially offering new hope to patients and healthcare professionals in the EU.
