Overview

• President Donald Trump previewed the rollout at an autism-focused event, calling it one of the most important announcements of his administration.
• The FDA approval, published in the Federal Register, specifies leucovorin for cerebral folate deficiency with immune markers such as anti–folate receptor alpha antibodies and includes orphan designation.
• Regulators asked manufacturers to pursue phase III studies measuring biomarkers, brain imaging and cognitive development to validate benefits and define responders.
• Early double-blind, placebo-controlled trials have reported gains in language and social communication for some children, and clinicians note benefits are likeliest in those who test positive for antifolate antibodies.
• Scientists caution the therapy is not a cure and report mostly mild side effects, while the administration’s promotion has drawn scrutiny given prior controversies involving HHS Secretary Robert F. Kennedy Jr.