The U.S. Food and Drug Administration has approved vepdegestrant for adults with advanced or metastatic ER-positive, HER2-negative breast cancer that carries ESR1 mutations, after the disease has progressed on prior endocrine therapy.
The approval is based on results from the Phase 3 VERITAC-2 trial, which compared vepdegestrant with fulvestrant. In patients with ESR1 mutations, the new drug improved progression-free survival to 5.0 months, compared with 2.1 months for fulvestrant. The response rate was also higher, at 19% versus 4%. However, in the overall study population, the benefit was minimal, with survival outcomes of 3.7 versus 3.6 months.
Common side effects included fatigue, nausea, anemia, and elevated liver enzymes. Serious side effects occurred in about 10% of patients, and only 3% stopped treatment بسبب side effects. The recommended dose is 200 mg taken orally once daily with food.
Experts say this approval offers an important new targeted treatment option for patients with ESR1-mutated breast cancer, a group that previously had limited choices after standard therapies stopped working.