May 18, 2015
An article published in Future Medicinal Chemistry presents an analysis of the orphan drugs classified as new molecular entities (NMEs) that were approved by the FDA between 1999 and 2012 for rare diseases. They found that out of the 102 orphan NMEs analysed, 46 were first in class – “discovered with phenotypic assays, target-based approaches and biologic strategies” –, 51 were followers of these drugs and five were imaging agents. In general their analysis demonstrated that “greater knowledge increases the chance of success and empirical solutions can be effective when knowledge is incomplete”.