Recent orphan drugs that are first-in-class

Abstract

Although the pipeline for new drugs seemed to be drying up, the number of new ‘first-in-class’ drugs appeared to be increasing. First-in-class drugs are those which use a new and unique mechanism for treating a disease or medical condition. They are a subset of new molecular entities (NMEs). The FDA reports that in 2012, 39 NMEs were approved. Twenty of these NMEs (∼ 50%) were identified by the FDA as first-in-class drugs. Of the 39 NMEs, 13 were designated orphan drugs and 9 of those orphan drugs were first-in-class. In 2013, there were 27 NMEs of which 9 were first-in-class. In 2013, numbers of NMEs are lower than the 2011 or 2012 but are based on fewer applications than in those years. There were 33% of NMEs that were first-in-class and 33% of the NMEs were orphan drugs. The FDA data indicate that creative new products are being developed for rare diseases.

Keywords::

• best-in-class
• FDA
• first-in-class
• mechanism
• me-too
• new molecular entity
• orphan drug