The FDA has approved a new use for Wellcovorin, or leucovorin calcium tablets, to treat cerebral folate deficiency, after initiating efforts to expand approval for autism-related symptoms.
The approval marks the first FDA-approved treatment for the rare genetic disease, which affects the transport of folate — a vitamin essential for brain health — into the brain, according to a March 10 news release from the agency. The drug is also approved to treat chemotherapy side effects.
Patients with folate receptor 1 gene-related cerebral folate deficiency can experience developmental delays, movement disorders, seizures and other neurological complications.
FDA Commissioner Marty Makay, MD, said the treatment may benefit some individuals who have “developmental delays with autistic features.”
However, the agency refrained from approving the medication as a treatment for autism. In September, President Donald Trump and Makay shared intentions to expand the drug’s approved use to treat some autism symptoms.
A senior FDA official said the agency did not have sufficient data to “establish the efficacy for autism more broadly,” NBC News reported.
The regulatory move comes as federal agencies increase research into potential “root causes” of autism.
