Novartis’s breast cancer drug Piqray (alpelisib) was the first new drug application for a new molecular entity approved under the US Food and Drug Administration (FDA) Real-Time Oncology Review (RTOR) pilot program, which aims to speed approvals while ensuring safety. The first targeted small-molecule kinase inhibitor against bladder cancer was approved with an accompanying companion diagnostic. A second drug with marginal efficacy in female sexual arousal disorder, the peptide Vyleesi, was approved (following Addyi in 2016). Phase 1/2 trials showed that an antisense oligonucleotide and an ex vivo lentiviral gene therapy produced responses in Huntington’s disease and Wiskott–Aldrich syndrome, respectively. Two new migraine drugs are in queue for decisions in the fourth quarter.