A relatively small number of people suffer from Huntington’s disease but it is an area of unmet medical need and one that could prove to be a valuable one for drugmakers, according to a new report from Datamonitor.

The study notes that firms have been reluctant to commit significant resources to drug discovery into HD. However, with a genetic test freely available for the disorder, “a shift in ‘big pharma’ thinking towards niche conditions and developments in the understanding of the disease”, Datamonitor’s central nervous system senior analyst Terence McManus says that drugmakers should look again “at what could be a lucrative market”.

HD is a highly genetically-linked disease, which is inherited in an autosomal dominant fashion. According to the US National Institute of Neurological Disorders and Stroke, at least 150,000 patients, have a 50% risk of developing HD in the six major markets for the disease – the USA, France, Germany, Spain, Italy and the UK. However, these people are not being treated properly, as current pharmacological therapy is limited to alleviating symptoms.

Datamonitor notes that antipsychotics are frequently used to partially suppress chorea (the signature movement disorder of the disease) and help control psychiatric abnormalities, such as agitation, hallucinations or psychotic delusions common in these patients. However, little scientific development has been achieved in symptomatic treatments over the last few years.

The report says that although Prestwick Pharmaceuticals’ tetrabenzine has recently been approved for HD patients by the US Food and Drug Administration, the treatment has been available in some European countries for a number of years. However there are other compounds in development.

Amarin Corp’s Miraxion is made from eicosapentaenoic acid, an omega-3 fatty acid that is currently in Phase III development, and the FDA has said that one additional (positive) late-stage trial, in conjunction with the confirmatory evidence from existing clinical data, may be sufficient to support a New Drug Application.

Medivation’s Dimbon is an orally-available small molecule that is believed to block the mitochondrial permeability transition pore (MPTP), the glutamate NMDA receptor and cholinesterase activity. The MPTP appears to be a new clinical target for tackling neurodegeneration and the drug is currently being investigated for HD in a Phase II trial. Elsewhere, Avicena’s HD-02 (ultra-pure creatine) may be a good candidate for prophylactic use, the report says, and incorporation into a pre-symptomatic patient’s diet as a food supplement may be viable.

Datamonitor concluded by saying research may prove to be a model for other neurodegenerative diseases “with much greater patient populations and commercial potential, such as Alzheimer’s and Parkinson’s disease”.