The US Food and Drug Administration (FDA) approved an almost record-setting 56 drugs in 2017, including December's first approval for an adeno-associated virus (AAV) gene therapy (Luxturna) and October's registration for a second chimeric antigen receptor T-cell (CAR-T) therapy (Yescarta). Promising results were reported for bluebird bio's lentiviral-based gene therapy in adrenomyeloneuropathy, two of four monoclonal antibodies (mAbs) against calcitonin gene-related peptide (CGRP) in migraine, and Vertex's combination of its small-molecule cystic fibrosis transmembrane regulator (CFTR) corrector therapy with Kalydeco.

Historic US regulatory approvals by lead indication

Notable clinical trial results (4Q17)

Catalysts (Q218)

Notable regulatory approvals (4Q17)

Notable regulatory setbacks (4Q17)