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.

Questions Answered:

  • What are the treatment drivers and goals for Dravet syndrome?
  • What attributes are key influences, which have limited impact, and which are hidden opportunities?
  • How do current therapies perform on key treatment drivers and goals for Dravet syndrome?
  • What are the prevailing areas of unmet need and opportunity in Dravet syndrome?
  • What trade-offs across different clinical attributes and price are acceptable to U.S. and European neurologists for a hypothetical new Dravet syndrome drug?

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

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