L1-79

L1-79 is an investigational oral small molecule drug developed for the treatment of core symptoms of Autism Spectrum Disorder (ASD). It is a first-in-class tyrosine hydroxylase inhibitor, specifically a racemic formulation of α-methylparatyrosine. By inhibiting tyrosine hydroxylase—the rate-limiting enzyme in the synthesis of catecholamines such as dopamine and norepinephrine—L1-79 modulates catecholaminergic signaling in the brain. Dysregulation of these pathways has been implicated in social and sensory difficulties characteristic of ASD. Clinical studies have shown that L1-79 can lead to statistically significant and clinically meaningful improvements in socialization and communication skills among adolescents and young adults with ASD, with a favorable safety profile. The drug received Fast Track designation from the FDA in 2018[1][2][4][5][8].

Other names

α-methylparatyrosine (racemic)racemic α-methylparatyrosine