Prime Highlights
- The S. Food and Drug Administrationhas approved Leucovorin as the first official treatment for Cerebral Folate Deficiency, a condition that prevents vitamin B9 from properly reaching the brain.
- Regulators said current scientific evidence does not support approving leucovorin for treating Autism Spectrum Disorder, despite interest from some families and researchers.
Key Facts
- Leucovorin, also called folinic acid, has been used for decades to reduce harmful side effects of chemotherapy.
- The approval applies to generic leucovorinand the older branded drug Wellcovorin, which GlaxoSmithKline previously sold.
Background
The U.S. Food and Drug Administration has approved the drug leucovorin as the first treatment for cerebral folate deficiency, a rare genetic disorder that affects how vitamin B9 reaches the brain.
Leucovorin, also known as folinic acid, is a synthetic form of vitamin B9. Doctors have used it for decades to reduce the harmful side effects of chemotherapy. With this new approval, doctors can now prescribe the drug specifically for children and adults with cerebral folate deficiency.
The decision follows earlier discussions about using leucovorin to treat symptoms related to autism spectrum disorder. Some families and researchers suggested that the drug could help children with autism improve language and social skills. However, regulators said the available evidence is not strong enough to approve it for autism treatment.
FDA officials explained that they began by reviewing research on leucovorin’s potential role in autism but later focused on patients with cerebral folate deficiency. According to the agency, this group showed the strongest scientific evidence and the most noticeable treatment benefits.
Cerebral folate deficiency is a rare condition that usually appears in children under the age of two. It occurs when the body cannot properly deliver folate to the brain. The disorder can lead to severe developmental delays, seizures, loss of muscle control and other neurological problems.
The approval is based on a review of existing medical research, including published case studies and scientific literature. While the review did not rely on large randomized clinical trials, regulators said the treatment results were strong enough to support approval.
The decision applies to both generic leucovorin and the older branded drug Wellcovorin, which was previously sold by GlaxoSmithKline.
The FDA has encouraged manufacturers to increase production to meet rising demand for the medication.
