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Beleodaq and Livatag, orphan drugs with blockbuster potential?

Onxeo is a company specializing in orphan drug oncology and currently has 3 products in late stage development. The orphan drug market, which is expected to grow to $176B by 2020 with a 2014-2020 CAGR of +10.5%, has attracted numerous biopharmaceutical companies seeking to get a share of this lucrative market. Despite the limited number of patients in orphan indications, the potential is huge as demonstrated by some orphan drugs that have become blockbuster drugs, generating more than $1 billion in sales per year. Onxeo's Beleodaq (belinostat) is a pan-HDAC inhibitor drug, currently marketed as second line treatment of Peripheral T-Cell Lymphoma, a rare form of Non-Hodgkin Lymphoma. Belinostat has a good tolerance and safety profile, and can be used in numerous cancer indications, as well as in combination therapy. Onxeo and its partner Spectrum Pharmaceuticals are currently evaluating which other indications they would further develop. This could significantly increase belinostat's market potential should they demonstrate efficacy. Onxeo also announced the 7^th positive DSMB for Livatag in primary liver cancer, currently in phase III. The Phase III preliminary results are expected in 2017.