Overview
- The FDA approval on Friday covers adults with ER‑positive, HER2‑negative advanced or metastatic breast cancer with ESR1 mutations after progression on endocrine therapy.
- Vepdegestrant is a PROTAC that targets the estrogen receptor for destruction rather than simply blocking it.
- The phase 3 VERITAC‑2 trial showed median progression‑free survival of 5.0 months with vepdegestrant versus 2.1 months with fulvestrant, reflecting a 43% lower risk of progression or death.
- The study also found a higher response rate with vepdegestrant at 19% versus 4% with fulvestrant, while overall survival data remain immature.
- Guardant360 CDx, a blood test that detects ESR1 mutations to guide use of the drug, also gained FDA clearance, and prescribing calls for 200 mg by mouth once daily with food with warnings for QTc prolongation and embryo‑fetal risk.