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New Therapies for Pulmonary Arterial Hypertension: Orphan Drugs Seeking Opportunities in Wider Related Indications
Datamonitor, July 2002
Pulmonary arterial hypertension (PAH) is a high value per patient niche market that until recently has been largely neglected in terms of the development of practical treatments. Several key advances over the past few months, including the launch of Tracleer and Remodulin, will play a major role in re-defining future treatment. With increasing awareness of the condition and several promising therapies in late stage development, treatment for PAH is set to enter a new era.Comprehensive analysis of the attractiveness of the PAH market based on epidemiology, unmet needs, orphan drug incentives and potential revenueIn-depth profiles of current treatments, including PAH specific and non-pah specific compoundsAnalysis of clinical trial data and market potential of the recently launched PAH specific therapies and compounds in late stage developmentDetailed comparative analysis of all PAH specific compounds in terms of efficacy, drug delivery, target audience and costEstimated potential of the PAH market and identification of opportunities of PAH specific drugs in wider related conditionsPAH is a rare condition with an estimated 100,000 sufferers across the US and EU. However, despite the low patient population, this market has attracted R&D investment due to the high unmet need and high value per patient of treatment. Orphan drug status and the potential for expansion of specific therapies into wider indications will continue to provide further investment incentives.Tracleer (bosentan) is the most promising PAH compound, although along with beraprost, it has had disappointing trial results in additional indications.In the future, new second generation endothelin receptor antagonists may offer superior benefits in both PAH and additional indications.Assess the attractiveness of the PAH market for R&D investment and/or in-licensing activityIdentify strengths, weaknesses, opportunities and threats of recently launched and late stage developmental therapiesIdentify opportunities and strategies for PAH specific compounds to be developed for additional indications
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