LGS is a rare and severe form of epilepsy that mostly affects individuals during infancy or early childhood. This syndrome has the tendency to develop between the age of one to eight years in children, and can be characterized by polymorphic seizures and neuropsychological decline. The cause of this syndrome can be symptomatic, which is secondary to an underlying brain disorder or it can be cryptogenic, which means the exact cause of the disease is unknown.
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In LGS, a number of affected children experience different types of seizures, with atonic, tonic, and atypical absence seizures being some common seizures experienced by the patients. Cognitive dysfunction, delay in reaching developmental milestones, and major behavioral problems in the children can be caused by this medical condition. LGS can be diagnosed on the basis of a thorough clinical evaluation, detailed history of patient, and a complete physical and neurological evaluation, including advanced imaging techniques.
Insights on Pipeline Segments
According to the research findings, majority of the drug candidates in the pipeline are being developed to be administered by the oral route. It has been observed that oral route of medications is convenient; available in delayed or rapid release formulation; provides improved patient’s compliance; has lesser risk of systemic infections; and is inexpensive in nature. Administration of therapeutics for LGS through oral route have shown promising results in the clinical studies. Also, maximum number of drugs in the pipeline are targeting GABA receptors and stimulating its mechanism of action.
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Positive Clinical Trial Results Act as a Stimulant for LGS Pipeline
The companies in the LGS therapeutics pipeline have shown positive clinical trial results in the various phases of drug development. For instance, in September 2018, Zogenix Inc, announced positive data from the Phase II trial of ZX008, for treatment of refractory patients with LGS. The results of the study concluded that ZX008 was generally well tolerated.
Globally, the Market Size for LGS Therapeutics is Expected to Increase Immensely in the next decade
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The global LGS therapeutics market is expected to advance at a CAGR of more than 10.0% during 2018-2028, mainly on account of increasing prevalence of the disease and growing interest of the companies to invest in the LGS pipeline. Among the various drugs that are currently approved for the treatment of LGS, Onfi is estimated to have the largest market share in 2018. Furthermore, other marketed drugs, including Topamax, Felbatol, Qudexy XR, and Epidiolex capture a very small share in the LGS therapeutics market.
Technological Advancements Play a Pivotal Role in the LGS Therapeutics Development
It has been observed that many pharmaceutical companies are leveraging new and improved technologies for the development of LGS therapeutics. These technologies play an important role in the drug development thereby helping the companies to develop more specific and effective targeted therapies. For instance, the focus of Aquestive Therapeutics Inc’s PharmFilm drug delivery technology platform and Supernus Pharmaceuticals Inc.’s Microtrol technology is to ensure faster onset of action, improved tolerability, lower dose exposures with equivalent efficacy, consistent, accurate dosing, ease of use, and improved adherence to medication over time.
Some of the key players involved in the development of LGS therapeutics in the late and mid stages of development include Zogenix Inc, Eisai Co. Ltd., Takeda Pharmaceutical Company Limited, and Marinus Pharmaceuticals Inc.
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