In the past year, the European Medicines Agency (EMA), the Food and Drug Administration
(FDA) and the Medicines and Healthcare Products Regulatory Agency (MHRA) authorised
53 novel drugs. While the 2024 harvest is not as rich as in 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 dynamic development of therapeutics
in areas of unmet need. The 2024 approvals of novel protein therapeutics (15) and
advanced therapy medicinal products (ATMPs, 6) indicate a sustained trend also noticeable
in the 2023 new drugs reviewed in this journal last year (16 and 11, respectively).
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 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 M 1 /M 4 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 authorised direct telomerase inhibitor (imetelstat) that reprogrammes the oncogenic
drive of tumour cells. In addition, an impressive percentage of novel drugs were first
in class (28 out of 53 or 53% of the total) and a substantial number can be considered
disease agnostic, indicating the possibility of future approved extensions of their
use for additional indications. 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.
