Carisbamate (DB12338): A Comprehensive Monograph on an Investigational Neuromodulator for Epilepsy

Section 1.0: Introduction to Carisbamate: An Investigational Neuromodulator

Carisbamate is an investigational, orally active, small molecule neuromodulator that has been the subject of extensive clinical research for various central nervous system (CNS) disorders, with a primary focus on epilepsy.[1] The developmental trajectory of this compound provides a compelling case study in pharmaceutical lifecycle management, characterized by a strategic repositioning following significant regulatory setbacks. Initially developed for a broad indication as an adjunctive treatment for partial-onset seizures, Carisbamate failed to secure marketing approval from major regulatory bodies. Subsequently, its development was redirected to target Lennox-Gastaut Syndrome (LGS), a rare, severe, and highly refractory form of childhood-onset epilepsy.[5]

Chemically classified as an organochlorine compound and an alkyl-carbamate, Carisbamate is structurally similar to felbamate, an antiepileptic drug (AED) approved in 1993.[5] Felbamate, while demonstrating broad-spectrum anticonvulsant activity, has its use severely restricted due to associations with fatal aplastic anemia and hepatotoxicity.[5] This chemical lineage immediately places Carisbamate's development within a critical context: the pursuit of novel carbamate-based therapies that retain anticonvulsant efficacy while offering a substantially improved safety profile.