FDA Approves Vepdegestrant: World’s First PROTAC Cancer Drug
Why in News?
The U.S. Food and Drug Administration (FDA) approved vepdegestrant (brand name: Veppanu) on 1 May 2026, making it the world’s first Proteolysis Targeting Chimera (PROTAC) drug. Developed jointly by Arvinas Operations Inc. and Pfizer Inc., the medicine is approved for treating ESR1-mutated, estrogen receptor-positive (ER+), HER2-negative advanced or metastatic breast cancer.
Vepdegestrant: Key Highlights
- Veppanu (Vepdegestrant) is an oral PROTAC therapy that degrades disease-causing proteins instead of merely blocking them.
- Approved for adults with ER-positive, HER2-negative breast cancer whose disease has progressed after at least one line of endocrine therapy.
- Patients must be identified through the FDA-approved Guardant360 CDx companion diagnostic, which detects ESR1 mutations.
Clinical Trial Findings
Approval was based on the Phase III VERITAC-2 Trial (NCT05654623).
- Median Progression-Free Survival (PFS): 5.0 months (Vepdegestrant) vs. 2.1 months (Fulvestrant).
- Objective Response Rate (ORR): 19% vs. 4%.
- Recommended dose: 200 mg orally once daily with food until disease progression or unacceptable toxicity.
Importance for Exams
- PROTAC (Proteolysis Targeting Chimera) is a new class of medicines that destroy harmful proteins through the body’s natural protein degradation system.
- Guardant360 CDx is an FDA-approved companion diagnostic used to identify patients with ESR1 mutations.
- Major safety warnings include QTc interval prolongation and embryo-fetal toxicity.
- The FDA approved the drug one month ahead of schedule under its Assessment Aid review process.
