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Huntington’s Disease Drug Development - Hope and Opportunity in an Active Pipeline
Decision Resources, Inc., June 2010, Pages: 32
Huntington’s disease (HD), a devastating neurodegenerative disease, is the subject of growing drug development activity as companies seek to address its significant unmet needs and capitalize on orphan-drug incentives. Increasing knowledge about the molecular mechanisms of HD and the characterization of a decades-long preclinical period have shaped a potentially lucrative drug market. Currently, sales of HD therapies in the United States are constrained by a small patient population, a paucity of effective therapies, and widespread use of generics, but thought-leading researchers and physicians contend that commercial opportunity awaits drug developers willing to forge a path toward disease-modifying therapies and novel agents for its most intractable symptoms.
Questions answered in this report:
- HD is a rare genetic disease affecting only a small percentage of the population. How does the prevalence of HD vary geographically? How do legislative incentives support commercial viability in this orphan disease market?
- Only one therapy is approved for HD, and so treatment is largely based on off-label regimens and has no demonstrated effect on HD’s relentless progression. How has the approval of Lundbeck’s Xenazine (tetrabenazine) affected the U.S. HD market? What opportunity remains for developers of novel symptomatic agents?
- HD is the subject of an active R&D pipeline characterized by novel approaches to modifying the disease’s progression or at least better controlling its symptoms. Are the potential benefits of disease-modifying approaches in HD patients proven or theoretical? What are thought leaders’ opinions about the directions of drug research in HD?
- Deficits in motor and cognitive function, increased behavioral problems, and neuropathological changes are now well documented in gene-positive HD patients before the onset of clinically manifest disease. Which organizations or companies have jumped into the untested waters of pre-HD clinical trials? What are the commercial opportunities that incentivize a paradigm shift in drug development?
Scope of the report:
- Primary research: Interviews with five HD experts in two countries.
- Patient population: General HD population in the United States and Europe; prevalence estimates for select regions worldwide.
- Potential HD etiology: Aggregation of mutant huntingtin protein, excitotoxicity, oxidative stress, mitochondrial dysfunction, and other mechanisms causing neuronal death.
- HD genetic basis, onset, and symptoms: Expression of mutant huntingtin protein; presentation of motor, cognitive, and behavioral symptoms; evolving characterization of prodromal/pre-manifest HD.
- Current and emerging HD therapies: Symptomatic treatments (including the only approved HD therapy, Lundbeck’s Xenazine); glutamate regulators, dopamine stabilizers, gene therapy, HDAC inhibitors, RNAi, antisense, and other emerging approaches.
- HD market size: 2009 U.S. HD pharmaceutical sales.
Key terms for this report:
ACR-16, Dimebon, Huntexil, Huntington Study Group, Huntington’s chorea, Huntington’s disease, Latrepirdine, Neurodegenerative disease, Neurology, Movement disorder, Orphan disease, Orphan drug, Pridopidine, Tetrabenazine, Xenazine
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