Post 03 November 2013

Rare Diseases R&D Pipeline

By definition, rare disease are those disorders which affect less than 200,000 patients in the US. A new report lists the more than 450 compounds under development by US companies for treatment of rare diseases.

The study was published by PhRMA, the Pharmaceutical Research and Manufacturers of America and can be downloaded at The range of rare diseases for which new drugs are being developing, is spanning from cancer (n=105 drug candidates), genetic disorders (n=85), neurological (n=32), infectious (n=28), respiratory (n=20) and autoimmune (n=18) diseases, to name the disease areas with most drug candidates in development.

The drug candidates in development for rare diseases are from all kind of product categories including vaccines, RNA, DNA, proteins, peptides, antibodies and, of course, small molecules.


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