Inherited Orphan Blood Diseases Patients Face Stagnant Treatment Future

Tuesday 29 January 2013, Amsterdam

Generally unimpressive treatment line-ups combined with weak product pipelines will lead to modest growth for several inherited orphan blood disease markets, states new market study.

According to the new report, the global therapeutic markets for sickle cell anemia, thalassemia and hereditary angioedema (HAE) are all expected to exhibit uninspired growth. The greatest value increase is expected from the sickle cell anemia market, which is forecast to climb at a Compound Annual Growth Rate (CAGR) of 9%, from US$36m in 2012 to US$70m in 2019.

Meanwhile, the global HAE therapeutics market is predicted to increase at a miserly CAGR of 3% during the same period, from US$1.5 billion to US$1.9 billion.

Kimberley Carter, Associate Analyst at GBI Research states: “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 ailments studied 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.

At present, only the HAE market can be considered strong, thanks largely 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.

This report provides insights into the up-and-coming trends of a portion of the inherited orphan blood disorder pharmaceutical market by examining sickle cell anemia, thalassemia and Hereditary Angioedema (HAE) in seven major markets.