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