Abstract
The US Food and Drug Administration approved 50 new drugs and nine new cellular and gene therapy products in 2024, i.e., a total of 59 new medical therapies. The latter group represented three treatments each for oncology and hematology/immunotherapy, and one each for neurology, genetic disorders, and cardiovascular disorders. Oncology, hematology/immunotherapy, and neurological disorders (14, six, and seven, respectively) also were highly prevalent among classic medications. Looking at trends over the past 5 years, we observe a greater share in first-in-class medications, more fast-track approvals, and mRNA/gene/cell-based therapies. While small molecules remain the largest fraction, their percentage has been declining substantially over the past 5 years. Taking together, these findings testify to the commitment of the pharmaceutical industry for innovative treatments, including conditions for which no approved therapies existed. On the other hand, there also is a trend for approvals for narrowly focused conditions such as tumors defined by genetic alterations.