Lennox-Gastaut syndrome: New treatments and treatments under investigation

May 11, 2020

Lennox-Gastaut syndrome: New treatments and treatments under investigation


a – Service de Neurologie Pédiatrique et des Maladies Métaboliques, CHU Hôpital Robert-Debré, 48, boulevard Sérurier, 75935 Paris Cedex 19, France
b – University Paris Diderot, inserm UMR1141, Paris, France Received 26 October 2019, Accepted 2 January 2020, Available online 11 May 2020.



There are currently five compounds (clobazam, felbamatelamotriginetopiramate, rufinamide) available for prescription with a demonstrated efficacy on drop seizures for Lennox-Gastaut syndrome (LGS). There are also currently new and under-investigation compounds. This paper gives an overview on these novel developments for LGS based on the lecture given at the French Chapter meeting of the International League Against Epilepsy (ILAE) held in Paris in October 2019. Five compounds were discussed. Epidiolex (cannabidiol) has been approved recently based on positive randomized control trials in LGS patients. Four drugs are under investigation according to a search on the ‘clinicaltrials.gov’ database. Perampanel and fenfluramine are both being studied in ongoing phase 3 studies. Two compounds are in an earlier stage of development with ongoing phase 1 and 2 studies: carisbamate and OV953. We summarized the publicly available data. Based on these drug development programs, we can expect that new compounds will become available for LGS in the next years, possibly resulting in new treatment paradigms.