Dravet syndrome (DS) is a rare, severe, pediatric-onset form of epilepsy characterized by developmental delay or regression and multiple seizure types. Only Diacomit (Biocodex’s stiripentol) is specifically approved for the adjunctive treatment of refractory DS (in Europe, Canada, and Japan), but the drug is difficult to access for U.S. prescribers and is not effective in all patients. Recognizing the clear unmet need for additional therapies specifically for DS, drug developers (e.g., GW Pharmaceuticals, Zogenix) are advancing new products through the pipeline that could offer important alternatives for this underserved orphan population. Understanding the drivers of clinical decision-making in DS and prescriber perceptions of today’s mostly off-label, generic options will help identify levers for new product positioning and differentiation.
Markets covered: United States, France, Germany, United Kingdom
Primary research: Survey of 60 U.S. and 30 European neurologists fielded in December 2016.
Key companies: Biocodex, GW Pharmaceuticals, Xenon Pharma, Zogenix
Key drugs: Diacomit (stiripentol), Onfi (clobazam), valproic acid, levetiracetam, topiramate, clonazepam, Fycompa (perampanel), Epidiolex, ZX-008