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Novel drugs approved by the EMA, the FDA and the MHRA in 2024: a year in review
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  • Stavros Topouzis,
  • Andreas Papapetropoulos,
  • Stephen Alexander,
  • Miriam Cortese-Krott,
  • David Kendall,
  • Kirill Martemyanov,
  • Claudio Mauro,
  • Nithyanandan Nagercoil,
  • Reynold Panettieri, Jr.,
  • Hemal Patel,
  • Rainer Schulz,
  • Barbara Stefanska,
  • Gary Stephens,
  • Mauro Teixeira,
  • Nathalie Vergnolle,
  • Xin Wang,
  • Peter Ferdinandy
Stavros Topouzis
University of Patras

Corresponding Author:stto@upatras.gr

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Andreas Papapetropoulos
University of Athens
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Stephen Alexander
Associate Professor in Molecular Pharmacology
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Miriam Cortese-Krott
Heinrich-Heine-Universität Düsseldorf
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David Kendall
University of Nottingham
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Kirill Martemyanov
UF Scripps Biomedical Research
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Claudio Mauro
University of Birmingham College of Medical and Dental Sciences
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Nithyanandan Nagercoil
Medicines and Healthcare products Regulatory Agency
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Reynold Panettieri, Jr.
University of Pennsylvania
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Hemal Patel
UC San Diego School of Medicine
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Rainer Schulz
Justus-Liebig-Universität Giessen
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Barbara Stefanska
University of British Columbia Faculty of Land and Food Systems
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Gary Stephens
University of Reading
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Mauro Teixeira
Universidade Federal de Minas Gerais
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Nathalie Vergnolle
Inserm U1220
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Xin Wang
The University of Manchester
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Peter Ferdinandy
University of Szeged
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Abstract

In the past year, the FDA, the EMA or the MHRA authorised fifty-three (53) novel drugs. While this 2024 harvest is not as rich as that of 2023, when 70 new chemical entities were approved, the number of “orphan” drug authorisations in 2024 (21) is similar to that of 2023 (24), illustrating the very dynamic development of therapeutics in areas of high unmet need. Clearly, the most striking characteristic of the 2024 drug yield is the creative pharmacological design, which allows these medicines to employ a novel approach to target a disease. Some such 2024 notable examples are: the first drug successfully using a ”dock-and-block” mechanism of inhibition (zenocutuzumab), the first approved drug for schizophrenia designed as an agonist of M1/M4 muscarinic receptors (xanomeline), the first biparatopic antibody (zanidatamab), binding two distinct epitopes of the same molecule, the first haemophilia therapy that instead of relying on external supplementation of clotting factors, restores Factor Xa activity by inhibiting TFPI (marstacimab), or the first ever authorized direct telomerase inhibitor (imetelstat) that reprogrammes the tumour cells’ oncogenic drive. In addition, in 2024 an impressive percentage of the novel drugs were first-in-class (28 out of 53, or 53% of the total) and a substantial number of them can be considered disease-agnostic, indicating the possibility of future approved extension of their use into additional indications. Overall, the 2024 harvest demonstrates the therapeutic potential of innovative pharmacological design, which allows the effective targeting of intractable disorders and addresses crucial, unmet therapeutic needs
03 Jan 2025Submitted to British Journal of Pharmacology
04 Jan 2025Submission Checks Completed
04 Jan 2025Assigned to Editor
04 Jan 2025Editorial Decision: Accept