FDA Approves Veppanu From Arvinas and Pfizer, the First Protein-Degrader Drug Cleared for Any Cancer
Vepdegestrant becomes the first PROTAC ever approved by the FDA, treating ESR1-mutated advanced breast cancer after one prior endocrine therapy.
Overview
The U.S. Food and Drug Administration on May 1, 2026 approved Veppanu (vepdegestrant), making it the first heterobifunctional protein degrader of its kind ever cleared by the agency, according to Arvinas. The drug, jointly developed by Arvinas and Pfizer, was approved for adults with estrogen receptor-positive (ER+), human epidermal growth factor receptor 2-negative (HER2-), 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.
The approval marks the first time the FDA has cleared a PROteolysis TArgeting Chimera (PROTAC), a type of heterobifunctional protein degrader, according to Arvinas.
A New Way to Drug a Target
As BioPharma Dive described the mechanism, the drug works by taking unwanted proteins — in Veppanu’s case, estrogen receptors — that are linked to disease and trashing them via the cell’s natural waste disposal system. That approach is distinct from conventional small-molecule inhibitors, which bind a target protein and block its activity rather than destroying it.
VERITAC-2 Trial Results
The approval was supported by the VERITAC-2 trial, a randomized study comparing vepdegestrant against fulvestrant. Among 270 patients whose tumors carried an ESR1 mutation, median progression-free survival was 5 months (95% CI: 3.7, 7.4) in the vepdegestrant arm versus 2.1 months (95% CI: 1.9, 3.5) in the fulvestrant arm, according to Arvinas.
The hazard ratio of 0.57 (95% CI: 0.42, 0.77; p=0.0001) translates to a 43 percent reduction in the risk of disease progression or death compared with fulvestrant, according to Arvinas. Vepdegestrant is taken orally once daily on a 28-day continuous dosing schedule, the company said.
The most common adverse events reported in the trial included decreased white blood cells, increased liver enzymes, musculoskeletal pain, fatigue, nausea, and decreased hemoglobin, according to Arvinas.
A Long-Awaited Class Reaches Patients
“This milestone demonstrates that targeted protein degradation can translate into meaningful clinical impact,” Randy Teel, chief executive officer of Arvinas, said in the company’s announcement. Noah Berkowitz, chief medical officer of Arvinas, added that “VEPPANU addresses an unmet need for patients with this aggressive form of breast cancer.”
Arvinas first announced its global collaboration with Pfizer on vepdegestrant in July 2021. In September 2025, the two companies said they planned to jointly select a third party to take over commercialization and potential further development of the drug, an arrangement that remains in place as Veppanu enters the market, the company said. BioPharma Dive reported that Arvinas shares climbed about 7 percent on news of the approval.
What It Means for the Field
Veppanu’s approval is narrow in scope — limited to a specific genetic subset of breast cancer patients who have already progressed on endocrine therapy. Even so, it provides the first regulatory validation that the protein-degradation approach can produce a marketable medicine, a milestone Arvinas highlighted as the first time a heterobifunctional protein degrader has cleared FDA review.