Year in Review: 2014 New and Noteworthy FDA Approvals (ABSTRACT)


Of the 54 notable drugs and biologics approved by the FDA in 2014, 20 are first-in-class agents for the management of cancers, cardiovascular risk, hepatitis C infection, insomnia, inflammatory bowel disease, leishmaniasis, lipodystrophy, mucopolysaccharidosis, onychomycosis, pulmonary fibrosis, and for immunization against serogroup B meningococcal disease.  Fourteen of these agents were approved under FDA’s "breakthrough therapy" designation reserved for drugs exhibiting superior efficacy against serious diseases. Trumenba,  a meningococcal group B vaccine, is the first new biologic agent to receive accelerated approval via this designation, along with four marketed drugs granted approval as breakthrough therapies for new indications: eltrombopag [Promacta], for cytopenias associated with aplastic anemia; ivacaftor (Kalydeco), for 8 rare gene mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene; and ibrutinib [Imbruvica] and ofatumumab [Arzerra], both for chronic lymphocytic leukemia. (CLICK FOR eCHAPTER 68 IN NEW eSECTION XI IN TOC