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Does the Treatment of Huntington's Disease Represent a Commercial Opportunity?
Decision Resources, Inc, August 2007, Pages: 22
Huntington’s disease (HD) is a genetic neurological disorder characterized by uncontrolled body movements and cognitive dysfunction. No drugs are approved to treat the disorder, leaving a high level of unmet need.
Commercial opportunity abounds, but the small patient population deters some companies. However, because of novel treatment approaches, interest in HD research is growing and companies may be able to tap into the enormous commercial potential, especially if a drug can treat multiple neurological diseases.
Questions Answered in This Spectrum Report:
-Huntington’s disease has a small patient population across all markets. What are the advantages and disadvantages for drug developers? What effect does orphan-drug status have on a drug’s potential?
-No therapies are approved specifically for HD; all current therapies are used off-label and provide only symptomatic relief. Which drugs are being investigated in clinical trials? What are the difficulties? When will a drug launch for HD?
-Novel treatment approaches including disease-modifying stem-cell and gene therapies are making some headway. What companies are investigating HD treatments and what are the most promising treatment approaches? At what stage are these treatments, and what are the chances of their launching for HD?
-Overview of Huntington’s disease: genetics, pathophysiology, epidemiology, symptoms, and diagnosis.
-Current treatment approach: no therapies are approved for HD; several drugs are used off-label to treat symptoms.
-Emerging therapies: disease-modifying agents, including Amarin’s Miraxion (Phase III) and Avicena’s HD-02 (Phase II), and symptomatic therapies, including Prestwick’s Xenazine (preregistered) and NeuroSearch’s ACR-16 (Phase II).
-Novel treatment approaches: stem-cell transplantation and gene therapy.
-Challenges and outlook: the future of the HD market.
Overview of Huntington’s Disease
The Genetics of Huntington’s Disease
Disease Onset, Symptoms, and Diagnosis
Epidemiology and Orphan Disease Status
Current Treatment Strategies
MethylGene and EnVivo’s Phenylbutyrate
Neurotrophic Factors: BDNF, GDNF, CNTF
Symptomatic Emerging Therapies
Prestwick Pharmaceuticals’ Xenazine
Sanofi -Aventis’s Rilutek
Challenges and Opportunities
1 Inheritance Patterns of Huntington’s Disease
2 Effect of CAG Repeats on Huntington’s Disease Development
3 Agents in Commercial Development for the Treatment of Huntington’s Disease
4 Agents Under Academic Investigation for the Treatment of Huntington’s Disease
1 Brain Structures Implicated in Huntington’s Disease
2 Prevalence of Huntington’s Disease in Select Geographic Regions
Novel Approaches to the Treatment of Huntington’s Disease: Stem-Cell Transplantation
Novel Approaches to the Treatment of Huntington’s Disease: Gene Therapy
-Depakene (valproic acid)
-Depakote (valproic acid and divalproex
-Prozac (fl uoxetine)