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  6. FDA approves vepdegestrant for ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer
  1. Resources for Information | Approved Drugs

FDA approves vepdegestrant for ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer

On May 1, 2026, the Food and Drug Administration approved vepdegestrant (Veppanu, Arvinas Operations, Inc.), a heterobifunctional protein degrader, for adults with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative, ESR1-mutated advanced or metastatic breast cancer, as detected by an FDA-authorized test, with disease progression following at least one line of endocrine therapy.

FDA also approved the Guardant360 CDx as a companion diagnostic device to identify patients with breast cancer with ESR1 mutations for treatment with vepdegestrant.

Full prescribing information for Veppanu will be posted on Drugs@FDA.

Efficacy and Safety

Efficacy was evaluated in VERITAC-2 (NCT05654623), a randomized, open-label, active-controlled, multicenter trial in 624 adults with ER-positive, HER2-negative, advanced or metastatic breast cancer, of whom 270 had tumors carrying ESR1 mutations. Patients were required to have disease progression on one to two lines of endocrine therapy, including one line with a CDK4/6 inhibitor. Patients were randomized (1:1) to receive vepdegestrant orally once daily, or fulvestrant intramuscularly on Days 1 and 15 of Cycle 1 and then once monthly thereafter. Randomization was stratified by ESR1 mutation status and visceral metastasis. ESR1 mutational status was determined by blood circulating tumor deoxyribonucleic acid (ctDNA) using central or local testing.

The major efficacy outcome measure was progression-free survival (PFS) as assessed by blinded independent central review (BICR) in the population of patients whose tumors had an ESR1 mutation and in the overall population. Additional efficacy outcome measures included overall survival (OS) and objective response rate (ORR) as assessed by BICR. In the population of patients whose tumors had an ESR1 mutation, a statistically significance difference in BICR-assessed PFS for vepdegestrant compared to fulvestrant was observed. Median PFS was 5 months (95% CI: 3.7, 7.4) in the vepdegestrant arm and 2.1 months (95% CI: 1.9, 3.5) in the fulvestrant arm (hazard ratio 0.57 [95% CI: 0.42, 0.77]; p-value 0.0001). ORR was 19% (95% CI: 12, 27) and 4% (95% CI: 1.6, 10) in the respective arms. OS was immature with 16% of deaths in this population at the time of the PFS analysis.

Warnings and Precautions

The prescribing information includes warnings and precautions for QTc interval prolongation and embryo-fetal toxicity.

The recommended vepdegestrant dose is 200 mg taken orally once daily with food until disease progression or unacceptable toxicity.

This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment. The FDA approved this application one month ahead of the FDA goal date.

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088.

For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact OCE’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.govOncProjectFacilitate@fda.hhs.gov.

Follow the Oncology Center of Excellence on X: @FDAOncology.

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