A year in pharmacology: new drugs approved by the US Food and Drug Administration in 2024

Aksoyalp Z. S., KAYKI MUTLU G., Wojnowski L., Michel M. C.

Naunyn-Schmiedeberg's Archives of Pharmacology, cilt.398, sa.5, ss.5077-5099, 2025 (SCI-Expanded) identifier identifier

  • Yayın Türü: Makale / Derleme
  • Cilt numarası: 398 Sayı: 5
  • Basım Tarihi: 2025
  • Doi Numarası: 10.1007/s00210-025-04020-2
  • Dergi Adı: Naunyn-Schmiedeberg's Archives of Pharmacology
  • Derginin Tarandığı İndeksler: Science Citation Index Expanded (SCI-EXPANDED), Scopus, BIOSIS, Biotechnology Research Abstracts, CAB Abstracts, Chemical Abstracts Core, Chimica, Veterinary Science Database
  • Sayfa Sayıları: ss.5077-5099
  • Anahtar Kelimeler: Cell-based therapy, FDA, First-in-class, Gene therapy, Monoclonal antibody, New drugs, Next-in-class, Orphan drugs, Rare diseases
  • Ankara Üniversitesi Adresli: Evet

Özet

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.