SCN8A Developmental and Epileptic Encephalopathy (SCN8A-DEE)
SCN8A-DEE is a rare disorder that causes a range of symptoms including severe epilepsy, developmental delay, and other medical challenges and is highly refractory to currently available antiseizure medication. SCN8A-DEE is linked to gain of function mutations in the SCN8A gene, a gene that affects how brain cells function. Mutations on this gene can cause neurological problems including epilepsy, intellectual disabilities, movement disorders and impaired development.
SCN8A-DEE is rare, with less than 1,000 patients believed to be diagnosed with the disorder worldwide. There are currently no approved therapies for this form of pediatric epilepsy.
NBI-921352: An Investigational Therapy for SCN8A-DEE
NBI-921352 is a potent, highly selective Nav1.6 sodium channel inhibitor being developed to treat pediatric patients with SCN8A developmental and epileptic encephalopathy (SCN8A-DEE).
Neurocrine Biosciences has received orphan drug and rare pediatric disease designations from the U.S. Food and Drug Administration (FDA) for NBI-921352 in SCN8A-DEE.
Neurocrine Biosciences acquired the exclusive rights to NBI-921352 from Xenon Pharmaceuticals.
Neurocrine Biosciences is also developing NBI-921352 for the potential treatment of focal-onset seizures.
Clinical Trials: SCN8A-DEE
The company is conducting the KAYAK™ study to assess the safety, tolerability, efficacy and pharmacokinetics of NBI-921352 as adjunctive therapy in patients with SCN8A-DEE. For more information about the study, please visit clinicaltrials.gov.