Anticipated Approval of Three New Therapies Through 2023 Will Expand Treatment Options for the Control and Relief of Motor Symptoms in Huntington’s Disease

June 12, 2014

Lack of Disease-Modifying Therapies is Expected to Remain a Critical Unmet Need and Provides Ample Opportunity for Developers, According to Findings from Decision Resources Group

BURLINGTON, Mass., June 12, 2014 /PRNewswire/ — Decision Resources Group finds that three therapies, Teva’s Huntexil, Auspex’s SD-809 and Raptor’s Procysbi, will likely receive regulatory approval for Huntington’s disease (HD) in the United States and the EU5 markets (Germany, France, Italy, Spain and United Kingdom) during the 2013-2023 study period. Notably, all three therapies are expected to be used primarily to control and relieve motor symptoms. Despite the very high unmet need for disease-modifying therapies in HD, such as gene therapies, none are expected to enter the market during the study period, owing to the considerable challenges of modulating gene expression in the human brain. Interviewed experts indicate awareness of the development of Isis Pharmaceuticals’ ISIS-HTT(RX) (an antisense oligonucleotide currently in preclinical studies) but acknowledge that serious barriers remain before this approach could provide real benefits to their patients.


Other key findings from the Niche Markets and Rare Diseases: Huntington’s Disease report:

    --  Opportunities for new product development: Because the unmet need in HD
        is so severe, regulators are likely to quickly approve a
        disease-modifying therapy offering even a modest reduction in disease
        progression. Interviewed neurologists agree that they would quickly
        embrace any therapy offering measurable improvements in loss of brain
        volume and/or reduction in cognitive impairment.
    --  Modest increase in the size of HD population: Driven by an aging
        population in the U.S. and Europe, the number of diagnosed prevalent
        cases of HD in the six major markets under study will increase by
        approximately 8 percent through 2023.
    --  Off-label use of antipsychotic drugs and antidepressants: Interviewed
        experts agree that antipsychotic drugs offer meaningful behavioral and
        hyperkinetic movement benefits for many of their HD patients. Physicians
        report that common adverse effects associated with antipyschotics, such
        as weight gain, sedation and extrapyramidal symptoms, can be beneficial
        in HD patients who struggle to control movements, maintain weight and
        achieve adequate rest. High levels of off-label prescribing of
        antipsychotic drugs and antidepressants will likely continue throughout
        the study period as no emerging therapies appear to offer significant
        behavioral or mood benefits at this time.

Comments from Decision Resources Group Analyst Jing Wu, M.S., M.B.A.:

    --  "HD is caused by mutations in a single gene and hence represents an
        ideal disorder to target with gene therapy. However, there are currently
        no available technologies to deliver and sustainably modulate gene
        expression in the brain neurons, and this critical challenge needs to be
        overcome before we see gene therapies developed for the treatment of
    --  "We expect Huntexil to launch before SD-809 (Auspex) and Procysbi in the
        U.S. and European markets, and to have good efficacy and tolerability;
        Teva will likely be able to leverage its long-established presence in
        the neurological diseases space to quickly drive uptake of Huntexil,
        which we expect will capture a greater patient share than the other two
        new therapies by 2023."

About Decision Resources Group

Decision Resources Group offers best-in-class, high-value information and insights on critical issues within the healthcare industry. Clients rely on this analysis and data to make informed decisions. Find out more at www.DecisionResourcesGroup.com.

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Decision Resources Group

Christopher Comfort



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SOURCE Decision Resources Group

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