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Dravet syndrome is a distressing infantile-onset epilepsy, which is sometimes accompanied by severe intellectual disabilities, autistic traits, hyperactive behavior, and ataxia. Discovery of SCN1A mutation in 2001 as a primary cause of this syndrome has accelerated the knowledge of the pathophysiological processes behind Dravet syndrome. SCN1A is responsible for encoding the ?-subunit of the voltage-gated sodium channel NaV1.1. Recent studies, using genetically modified mouse models, have indicated that haploinsufficiency of NaV1.1 in GABAergic inhibitory interneurons in the forebrain may be involved in the fever sensitivity of seizures, seizure susceptibility, autistic traits, and premature death characteristics of Dravet syndrome. In recent years, human cellular models of Dravet syndrome have been recognized by the use of induced pluripotent stem cells (iPSCs) derived from patients. Functional susceptibilities in GABAergic neurons have been discovered by studying these human disease models.
Increasing R&D into new drug development for Dravet syndrome and approval for these drugs in key markets, such as, North America and Europe are anticipated to propel the development of the Dravet syndrome treatment market worldwide. For instance, in February 2018, Zogenix Inc. revealed U.S. Food and Drug Administration (FDA) approval of its Phase III drug ZX-008 for seizures associated with Dravet Syndrome. In 2017, Epygenix Therapeutics, Inc., a biopharmaceutical company, received FDA orphan drug designation for its product, EPX-300, indicated for the treatment of the Dravet syndrome. In 2017, Ovid Therapeutics (the U.S.) got orphan drug designation from FDA for its product, TAK-935/OV935. Furthermore, collaborations among key market players for the development and manufacturing of drugs indicated for the treatment of Dravet syndrome are expected to drive the growth of the Dravet syndrome treatment market, globally. For instance, in 2017, Epygenix Therapeutics, Inc. and, Laurus Synthesis Inc. entered into a strategic collaboration for the development and manufacturing of EPX-100.
A recent U.S. epidemiology study concluded that Dravet syndrome incidence has increased to twice the previous year, affecting 1:15,700 infants, 80% of whom have an SCN1A mutation. Unfortunately, a large number of children do not get access to early diagnosis, which could have had a significant impact on long-term development and quality of life. The study concluded that genetic testing via an epilepsy panel should be considered in children with two or more prolonged seizures by the age of one year.
The Dravet syndrome treatment market can be segmented based on treatment type, distribution channel, and geography. By treatment type, the market can be divided into anticonvulsant, benzodiazepines, vagal nerve stimulation, and others. In terms of distribution channel, the marekt can be categorized into hospital pharmacies, retail pharmacies, and online pharmacies.
On the basis of geography, the global Dravet syndrome treatment market can be divided into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America held the leading share of the global Dravet syndrome treatment market in 2017, followed by Europe, due to growth initiatives and funding by various organizations to support the development of new treatment for Dravet syndrome. For instance, in 2017, Dravet Syndrome Foundation, a non-profit organization of the U.S., funded an amount of US$ 3,600,000 for the development of new treatment methods for the disorder. The Dravet syndrome treatment market in Europe is likely to exhibit significant growth during the forecast period due to increasing research and development activities conducted by key market players in the region. For instance, a British biopharmaceutical company, GW Pharmaceuticals, developed Epidiolex (cannabidiol) ingredient derived from marijuana. The company submitted a new drug application (NDA) for Epidiolex to the U.S. FDA in December 2017. After approval, the drug is expected to hit the U.S. market by mid of 2018.
Key players operating in the Dravet syndrome treatment market include Epygenix Therapeutics, Inc., GW Pharmaceuticals Plc., Ovid Therapeutics, Takeda Pharmaceutical Company Ltd, OPKO Health Inc., Zogenix, Inc, Biocodex S.A., PTC Therapeutics, Inc., and Biscayne Neurotherapeutics, Inc. Key players in the market are focusing on obtaining approval for their new drugs from major regulatory bodies, such as, FDA and European Commission.
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