New FDA-Approved Targeted Therapy for ESR1-Mutated Metastatic Breast Cancer

The FDA has approved vepdegestrant (Veppanu), an oral therapy for adults with ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer whose disease has progressed after prior endocrine therapy.

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The FDA has approved a new targeted treatment option for certain people living with advanced breast cancer.

What was approved?

  • The FDA approved vepdegestrant (Veppanu) for adults with:
    • Estrogen receptor (ER)-positive breast cancer
    • HER2-negative breast cancer
    • An ESR1 mutation in their cancer
    • Advanced or metastatic disease that has worsened after at least one prior endocrine (hormone) therapy, including a CDK4/6 inhibitor.
  • Vepdegestrant is a type of oral “protein degrader,” designed to target and break down the estrogen receptor inside cancer cells.

The FDA also approved a blood test, Guardant360 CDx, to find ESR1 mutations and help identify who may benefit from vepdegestrant.

How does this treatment perform?

In the VERITAC‑2 clinical trial, researchers compared vepdegestrant taken once daily by mouth with fulvestrant, an older anti-estrogen drug given as an injection.

Among people whose tumors had an ESR1 mutation:

  • Those who received vepdegestrant went a median of about 5 months before their cancer grew or spread again.
  • Those who received fulvestrant went a median of about 2.1 months before their cancer progressed.
  • More people had their tumors shrink on vepdegestrant (about 19%) compared with fulvestrant (about 4%).
  • Overall survival results are still early and will continue to be followed over time.

These findings suggest that vepdegestrant may help people with ESR1‑mutated, ER‑positive, HER2‑negative metastatic breast cancer stay in control of their disease longer than with fulvestrant alone.

About Vepdegestrant

Vepdegestrant (Veppanu) is a type of hormone-targeted cancer medicine used to treat certain kinds of advanced or metastatic breast cancer that are estrogen receptor (ER)-positive and HER2-negative. It works by finding and breaking down the estrogen receptor inside cancer cells, which can help slow or stop the signals that tell these cells to grow. Vepdegestrant is taken as a pill by mouth, which may feel more convenient than injections for some people. Not everyone with breast cancer will benefit from this drug, so your care team may recommend special testing to see whether your tumor has an ESR1 mutation and whether this medicine is a good option for you.

What this could mean for patients

For people whose ER‑positive, HER2‑negative metastatic breast cancer has progressed after prior hormone therapy and a CDK4/6 inhibitor, and whose tumors carry an ESR1 mutation, vepdegestrant offers a new, targeted oral option.

If you or a loved one is living with advanced breast cancer, you may want to ask your oncology team:

  • Has my tumor been tested for ESR1 mutations?
  • Am I a candidate for vepdegestrant (Veppanu) based on my prior treatments and test results?
  • Is Guardant360 CDx or another ESR1 test appropriate for me now?

Reference

https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-vepdegestrant-er-positive-her2-negative-esr1-mutated-advanced-or-metastatic-breast?utm_medium=email&utm_source=govdelivery

Campone M, De Laurentiis M, Jhaveri K, et al; VERITAC-2 Study Group. Vepdegestrant, a PROTAC Estrogen Receptor Degrader, in Advanced Breast Cancer. N Engl J Med. 2025 Aug 7;393(6):556-568.

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