Huntington’s Disease | Niche & Rare Disease Landscape & Forecast | US/EU5 | 2016

Huntington’s disease (HD) is a neurodegenerative genetic disorder caused by an abnormal trinucleotide repeat in the Htt gene. Aggregation of the product of the mutant allele, mHtt, drives the progressive loss of caudate and striatal neurons, resulting in chorea, various behavioral problems, cognitive impairment, and in some cases, dementia. Treatment of HD consists of symptomatic management, and no DMT is yet available. Chorea is treated by tetrabenazine or various atypical antipsychotics, while mild depression, irritability, and anxiety are usually treated with SSRIs. Mood-stabilizing antiepileptics and benzodiazepines also see some use in patients whose symptoms are not adequately controlled by antidepressants or antipsychotics. At present, no therapy exists for the management of cognitive impairment in HD. Key emerging agents in the HD pipeline include Teva’s SD-809, a deuterated form of tetrabenazine that offers dosing and tolerability improvements over tetrabenazine; and Teva’s pridopidine, a dopamine stabilizer being developed for HD motor symptoms that may also have disease-modifying activity. The early-stage HD pipeline is diverse with respect to agents’ mechanisms of action, but is focused almost exclusively on motor dysfunction. The relative lack of cognitive therapies and potential DMTs in the HD pipeline leave tremendous market potential for drug development in this incurable, fatal disease.

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