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"Orphan Disease Therapeutics in Genetic Disorders to 2018 - Emerging Agents in Cystic Fibrosis Offer Strong Opportunities for Investment and Licensing Activity" - New Market Report

Recently published research from GBI Research, "Orphan Disease Therapeutics in Genetic Disorders to 2018 - Emerging Agents in Cystic Fibrosis Offer Strong Opportunities for Investment and Licensing Activity", is now available at Fast Market Research


Boston, MA -- (SBWIRE) -- 06/08/2012 -- GBI Research, the leading business intelligence provider, has released its latest research, "Orphan Disease Therapeutics in Genetic Disorders to 2018 - Emerging Agents in Cystic Fibrosis Offer Strong Opportunities for Investment and Licensing Activity", which provides insights into the global orphan diseases in genetic disorders therapeutics market, including market forecasts until 2018. The report provides an in-depth analysis of major orphan genetic disease indications, covering Cystic Fibrosis (CF), Duchenne Muscular Dystrophy (DMD), Fabry disease and Pompe disease. The report also includes insights into the orphan genetic disease therapeutics R&D pipeline. It analyzes the competitive landscape, including M&As, and licensing and co-development deals. The report is built using data and information sourced from proprietary databases, primary and secondary research and in-house analysis by GBI Research's team of industry experts.

View Full Report Details and Table of Contents

GBI Research found that the global orphan disease therapeutics in genetic disorders market was worth $1.5 billion in 2010, having grown from $559m in 2004 at a Compound Annual Growth Rate (CAGR) of 17.3%. It is expected to increase to $4.1 billion by 2018, growing at a CAGR of 13.8% over the period from 2010. This high rate of growth in the historic period can be attributed to the entry of novel, highly priced therapies into markets with high unmet need. These include Myozyme for Pompe disease and enzyme replacement therapies for Fabry disease, which have high costs of over $200,000 per patient per year. More highly-priced therapies are expected in the forecast period, to further drive growth. The recent approval of Kalydeco in CF and the expected approval of exon-skipping therapies for DMD are examples of this. Competitive activity is also increasing as larger companies realize the rewards of investing in orphan therapeutics, and the high unmet need and low competition in many markets is expected to attract more into the space in the future.


- Data and analysis on the global orphan disease therapeutics in genetic disorders in the leading geographies of the world- the US, the UK, Germany, France, Italy, Spain, and Japan.
- Annualized market data for the orphan disease therapeutics in genetic disorders market from 2002-2010 with forecasts to 2018.
- Market data on the therapeutic landscape, covering CF, DMD, Fabry disease and Pompe disease. This includes market size, market share, annual cost of therapy, treatment flow algorithm and branded and generic share.
- Key drivers and restraints that have had a significant impact on the market and on each indication.

Companies Mentioned in this Report: GlaxoSmithKline, Prosensa, Santhera, AVI BioPharma, Genzyme (Sanofi Subsidiary), Shire, PTC Therapeutics, Novartis, Genentech (Roche), Gilead

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