EveryONE Medicines, a biotech startup focused on personalized treatments for rare diseases, has decided to cease operations. According to an anonymous source cited by Endpoints News, the company found the U.S. Food and Drug Administration’s (FDA) recent guidance on bespoke medicines inadequate for supporting commercialization efforts targeting very small patient populations.
The FDA recently released draft guidance aimed at helping drug developers advance personalized genetic medicines. The recommendations suggest that companies should identify the biological cause of a disease and design their therapies to address this specific pathway. The agency also noted that companies could use well-documented natural history data from untreated patients as controls and show clear improvements in clinical outcomes.
However, the framework does not include a process-based approval system. In contrast, health regulators in the United Kingdom have implemented a similar pathway that allows for “process-level approvals.” This approach enables easier regulatory submissions for different drugs developed using the same approved processes. EveryONE had previously received such approval in the U.K., which facilitated its drug testing and manufacturing operations.
Under the FDA’s current framework, each individualized medicine must be submitted as a separate application with unique documentation—a requirement that can be challenging for smaller firms like EveryONE.
EveryONE CEO Julia Vitarello declined to comment on the company’s closure but stated: “With the new FDA pathway, the industry is in ‘a significantly better place than we were even a year ago.’” Vitarello founded EveryONE in 2024 after her daughter died from Batten disease, a rare neurological disorder. The company aimed to create standardized pathways for developing individualized medicines for small patient groups.
Andrew Nixon, spokesperson for the Department of Health and Human Services, responded to concerns about the new regulatory approach: “Any suggestion that FDA’s individualized therapy framework caused a private company’s shutdown is baseless and would be completely false reporting,” Nixon told Endpoints.
BioSpace has contacted EveryONE Medicines for further comment.
