GlobalData, a global commercial intelligence provider, expects the Huntington’s disease (HD) market to show a compound annual growth rate of 23.9% in the United States between 2014 and 2024. By 2024, it is anticipated that the U.S will account for approximately 77% of total HD drug sales among the seven major global markets (i.e., the U.S., the 5EU countries [France, Germany, Italy, Spain, and the United Kingdom], and Japan).
HD is a rare, autosomal-dominant, neurodegenerative disorder characterized by impaired motor control, cognitive dysfunction, behavioral changes, and mood disorders.
GlobalData expects several factors to drive growth in the HD market during the 10-year forecast period:
Currently, the global HD market is dominated by symptomatic treatments, with only one drug––the catecholamine-depleting agent tetrabenazine (Xenazine, Lundbeck)––approved for the treatment of HD-associated chorea in the U.S., according to the GlobalData report. The remaining symptomatic treatments, including antipsychotics, antidepressants, benzodiazepines, anticonvulsants, antiparkinson drugs, and botulinum toxin, are used off-label for this indication.
The development of a DMD for the treatment of HD is the most significant unmet need, the report points out. Drug developers are now moving beyond symptom-alleviating therapies, and numerous potential DMDs for HD patients are undergoing clinical development.
GlobalData has identified four drugs with potential disease-modifying properties that are in late-stage clinical development for HD: RP103 (Raptor Pharmaceuticals), a delayed-release formulation of the cystine-depleting agent cysteamine bitartrate; PBT2 (Prana Biotechnology), a second-generation 8-hydroxyquinoline analog; the immunomodulator laquinimod (Teva Pharmaceuticals), and PF-02545920 (Pfizer), a phosphodiesterase-10 inhibitor. All of these drugs are proposed to have different mechanisms of action, with the potential to be used for the treatment of all HD patients once they reach the market. RP103 is expected to come to market first, giving this drug an important advantage over the other DMDs in the late-stage pipeline, according to the report.
Several potential DMDs are also in early-stage clinical development for HD. GlobalData found that key opinion leaders are particularly interested in two of these agents, the Gen. 2.0+ antisense drug IONIS-HTTRx (Ionis Pharmaceuticals) and the monoclonal antibody VX-15/2503 (Vaccinex).
During the 10-year forecast period covered by the report (2014–2024), it is expected that four new HD treatments will launch in the U.S. and 5EU markets. SD-809 (Teva), a small-molecule inhibitor of vesicular monoamine 2 transporter (VMAT2), is currently in the preregistration phase in the U.S. This drug is a symptomatic treatment with the same mechanism of action as that of currently marketed tetrabenazine. SD-809 has a more favorable safety profile, however.
Pridopidine (Teva) is a first-in-class D2 receptor stabilizer designed for the symptomatic treatment of nonchoreic motor symptoms in HD patients. The drug is expected to be the second pipeline agent to launch in the U.S., 5EU, and Japan.
GlobalData expects that RP103 and PBT2 will launch only in the U.S. and 5EU during the forecast period. Both drugs are potential first-in-class DMDs, and since DMDs are highly anticipated for this indication, they are likely to gain a large market share after their launch, the company predicts. Throughout the forecast period, it is expected that either RP103 or PBT2 will be administered in combination with symptomatic treatments, competing only with each other and not with any other drugs or drug classes included in the forecast.
Source: GlobalData; May 2016.