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Amyotrophic Lateral Sclerosis - Orphan Status, Unmet Need, and Diverse Drug Targets Offer Opportunities for Drug Developers
Decision Resources, Inc, Feb 2011, Pages: 32
According to experts interviewed by Decision Resources, a strong unmet need exists for new agents to treat amyotrophic lateral sclerosis (ALS); physicians and patients would easily adapt to any new agent that could significantly inhibit disease progression. Only one drug has been approved for ALS, and it has only a modest effect on survival time of patients suffering from this fatal disease. Given this lack of available treatments, as well as the disease’s orphan-drug status - which provides numerous financial, marketing, and drug-approval benefits - substantial opportunity awaits a company that develops an efficacious agent to treat this disease.
Questions answered in this report:
- ALS affects predominantly the elderly population, with most cases occurring in those aged 50 or older. What is the overall prevalence by gender and age? What is the predicted rate of growth in prevalent cases of ALS over a ten-year forecast period?
- Sanofi-Aventis’s Rilutek (riluzole) is the only approved treatment that slows the progression of ALS. What is the outlook for Rilutek? Which companies have drugs in late-stage development for ALS? Is Sanofi-Aventis’s position threatened by promising emerging therapies?
- Several drug candidates are in various stages of development for ALS treatment. What is the mechanism of action of these drugs? Do they target different pathways? What is the view of interviewed neurologists on drugs in the ALS pipeline?
- ALS is a rare disease; it affects a small percentage of the population and qualifies as an orphan disease. How does the U.S. government encourage the pharmaceutical industry to investigate treatments that qualify for orphan status? What are the advantages of developing an orphan drug?
- Most other current therapies for ALS patients are used for symptomatic treatment and palliative care. What are the current symptomatic treatments for ALS patients? How many such agents have been approved by the FDA for ALS? Are any emerging symptomatic agents in the pipeline for ALS? What is the opinion of interviewed experts on these symptomatic treatment(s) for ALS?
- One reason for the caution about emerging agents is the number of agents that, despite early promise, have ultimately failed to offer positive benefit to ALS patients. What emerging agents have recently failed large clinical trials? What are the challenges in running large-scale clinical trials for ALS?
Scope of the report:
- Market covered: United States
- Primary research: Interviews with thought leaders.
- Epidemiology: Prevalent U.S. cases over a ten-year forecast period; age and gender trends.
- Mechanism of action of emerging agents: Antiglutamatergics, heat shock inducers, antioxidants, neuroprotectives, antiapoptotics, vascular endothelial growth factor (VEGF) inducers.
- Emerging therapies: Phase I: 9 drugs; Phase II: 14 drugs; Phase III: 9 drugs.
- Exhibits: 15 tables and figures.
- Clinical trial considerations: New diagnostic criteria and potential modifications for enrolling in future ALS clinical trials.
- Outlook for ALS: Sales of Rilutek and considerations for drug developers.
- Benefits of orphan-drug status: Financial and marketing incentives associated with orphan-drug status.
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