While an anonymous source tied the closure to shortcomings in the FDA’s new pathway, a spokesperson for the Department of Health and Human Services pushed back on the suggestion.
EveryONE Medicines has decided to close up shop. An anonymous source told Endpoints News that the biotech found the FDA’s recent bespoke framework for personalized medicines insufficient and challenging to work with to commercialize treatments for exceedingly small patient populations, the outlet reported on Tuesday.
BioSpace has reached out to EveryONE for comment.
The regulator last week released draft guidance designed to help drugmakers that are advancing personalized genetic medicines, potentially clearing an easier path to market. Under these new recommendations, the FDA has suggested that companies should clearly elucidate the underlying biologic cause of a disease and develop their drug candidate to target this pathway.
As a reference for their program’s efficacy, drugmakers can use well-characterized natural history untreated controls and demonstrate clear improvements in clinical outcomes, the FDA added. Missing from the regulator’s framework, according to Endpoints’ source, is a process-based approach to approvals.
In the U.K., which last year released a similar pathway, health regulators can grant “process-level approvals,” which can enable the development of “platform-based technologies and individualized medicines.” Under this approach, EveryONE received approval for its drug testing and manufacturing processes, allowing for easier regulatory submissions for different drugs that use these same processes, according to Endpoints.
Under the FDA’s framework, however, each individualized medicine will be treated as a discrete application, with a submission package built from the ground up each time—a burden that can prove too heavy for smaller companies like EveryONE.
EveryONE CEO Julia Vitarello declined to comment to Endpoints about the company’s closure and said that with the new FDA pathway, the industry is in “a significantly better place than we were even a year ago.” Vitarello launched EveryONE in 2024 after her daughter lost the battle against Batten disease, a rare and fatal condition that manifests as a constellation of neurological symptoms. The biotech focused on creating standardized, scalable pathways for individualized medicines to enable the development of drugs for diseases that affect small patient populations.
Andrew Nixon, spokesperson for the Department of Health and Human Services, insisted that the industry has been strongly supportive of the new plausible mechanism pathway.
“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.
