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Inherited Orphan Blood Diseases Patients Face Stagnant Treatment Future

By BiotechDaily International staff writers
Posted on 28 Jan 2013
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SARTORIUS AG
Uninspiring treatment options combined with feeble product pipelines typically will lead to modest growth for several inherited orphan blood disease markets, according to recent market research.

According to the business intelligence provider GBI Research’s (New York, NY, USA) new report, the global therapeutic markets for sickle cell thalassemia, sickle cell anemia, and hereditary angioedema (HAE) are all expected to show unimpressive growth. The greatest value increase is expected from the sickle cell anemia market, which is forecast to rise at a compound annual growth rate (CAGR) of 9%, from USD 36 million in 2012 to USD 70 million in 2019.

In the meantime, the global HAE therapeutics market is predicted to increase at a miserly CAGR of 3% during the same period, from USD 1.5–1.9 billion. Kimberley Carter, associate analyst at GBI Research, stated, “Even though orphan blood disorders are rare, the three diseases featured in this report are hereditary and have a large impact on the lives of the families and ethnic groups afflicted by these diseases. Many of these diseases cause debilitating health issues and limit lifespan. Because of this, there is a great need for curative and disease modifying therapies. While this may require significant R&D investment, any drugs that are successful in treating these diseases can be expected to be priced at the highest premium range.”

All three disorders examined in the new report have weak product pipelines, with most upcoming treatments either too similar to currently available options, or insufficiently promising to make any real impact.

Presently, only the HAE market can be considered strong, due mostly to the dominance of Cinryze—the only branded therapy taken according to a regular dosing regimen. Cinryze will continue to dominate the HAE market as long as it remains unique, and an exceptionally weak pipeline will most likely ensure this remains the case.

GBI Research is a provider of business intelligence reports, providing data and forecasts based on the insights of key industry leaders to ensure up-to-date with the latest emerging trends in markets.

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