Orphan blood disease therapeutics market: Myelofibrosis to be fastest growing sector by 2019

Press Release   •   Feb 26, 2013 12:10 GMT

Our report on the orphan blood disease therapeutics market reviews and forecasts the five acquired orphan blood diseases up to 2019. The indications covered are Paroxysmal Nocturnal Hemoglobinuria (PNH), Idiopathic Thrombocytopenic Purpura (ITP), Myelodysplastic Syndrome (MDS), Myelofibrosis (MF) and Polycythemia Vera (PV). As orphan diseases, they have very low prevalence and incidence rates and in many cases may disproportionately affect individuals of a particular age or sex. Many orphan diseases have high unmet clinical needs and premium pricing potential for successful new market entries; this is the case for all five indications covered.

The MF market will be the fastest growing of the five markets during the forecast period, at a Compound Annual Growth Rate (CAGR) of 34% and reaching a total market size of $1,127m in 2019. MDS represents the largest market, expected to reach $2,251m by 2019.

The PNH treatment market in the US, the top five EU countries (the UK, France, Germany, Italy and Spain), and Japan is expected to grow at a Compound Annual Growth Rate (CAGR) of 4% over the 2011–2019 period to $1,164m by 2019.

These five indications offer significant treatment challenges and many patient populations have a high level of unmet need, in turn offering diverse examples of drug markets.

The PNH market is dominated by Soliris, which is currently the world's most expensive drug. Due to its market dominance, the PNH pipeline is very weak. The outlook for the MDS market is good as it has the largest pipeline of all five indications. This is also boosted by improving disease diagnosis and awareness rates. Similarly, MF, a disease with significant unmet needs for treatment, also has a promising pipeline and improving diagnosis rates.

Jakafi (ruxolitinib) is a JAK inhibitor which works to target the genes believed to cause MF, approved by the FDA in November 2011 for the treatment of intermediate or high-risk MF, and perhaps set to be a future blockbuster drug.

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