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Aplastic Anemia Patients in Need of New Treatment Options

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Significant opportunity exists for new drug entrants offering improved effectiveness and safety in the treatment of aplastic anemia, as existing products are associated with serious complications and relapses, a new report by GlobalData has found.

The new study explores the failings of current treatment options for aplastic anemia, which include allogeneic stem cell transplant and immunosuppressive therapies treatments primarily dominated by generics.

These therapies are only effective in approximately 70% of patients, and stem cell transplant is associated with risks of graft failure and graft-versus-host disease (GVHD).

Risk of relapse and further development of myelodysplastic syndrome (MDS) also limits the use of immunosuppressive therapy. Therefore, the aplastic anemia therapeutics market offers significant opportunity for new therapies able to offer reduced risks and superior effectiveness.

However, GlobalData's analysis of the drug pipeline shows an absence of any promising, industry-sponsored molecule in a late stage of development.

The only molecule in Phase II/III is alemtuzumab, which is under investigation as a conditioning regimen for patients with severe aplastic anemia undergoing allogeneic stem cell transplantation using matched unrelated donors and mismatched related donors.

As this represents a very small percentage of patients, the drug will therefore have a limited effect on the therapeutics market.

Eltrombopag provides the only potentially significant breakthrough for aplastic anemia patients and is expected to be the driving force behind the future growth of the aplastic anemia market.

However, Eltrombopag is only currently under Phase II of development, with a trial initiated in March 2011, and launch is not expected until at least 2018.

As a result of these factors, GlobalData suggests that the aplastic anemia market will show slow growth in the future scenario, developing at a negligible Compound Annual Growth Rate (CAGR) of 1.1% from $73.3m in 2011 to $79.7m by 2019.