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.