FDA approves vepdegestrant, marking major shift in cancer therapy

Washington: The U.S. Food and Drug Administration has approved vepdegestrant, the world’s first therapy based on PROTAC technology, in what experts call a major shift in how drugs target disease.

On May 1, the FDA cleared vepdegestrant for patients with ESR1-mutated, ER-positive, and HER2-negative advanced breast cancer. The approval is the first by the FDA — whose decisions are widely followed by regulators worldwide — for a drug built on PROTAC technology.

Unlike traditional drugs that block harmful proteins, PROTACs are designed to remove them from cells entirely. “This paves the way for drugs designed to remove harmful proteins from cells, rather than simply block them,” researchers noted.

The advance is significant because PROTACs can sidestep long-standing barriers to treating diseases involving “undruggable” proteins — targets that conventional medicines have failed to reach. Scientists have been developing the technology for more than two decades.

Vepdegestrant’s approval is expected to accelerate development of PROTAC drugs for other cancers and diseases driven by problematic proteins.