Top Selling (2026) Small Molecule Orphan Drugs: A Journey into Their Chemistry

Tiz, Davide Benedetto; Bagnoli, Luana; Rosati, Ornelio; Marini, Francesca; Sancineto, Luca; Santi, Claudio

doi:10.3390/ijms24020930

Cited by 2 publications

(1 citation statement)

References 47 publications

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“…In this context, the incentives provided by the FDA also drive greater resource allocation to these diseases. Annual growth in the development of Orphan Drugs is now expected [17,18].…”

Section: Orphan Drugsmentioning

confidence: 99%

FDA Approvals of Biologics in 2022

2023

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The year 2022 witnessed the control of the COVID-19 pandemic in most countries through social and hygiene measures and also vaccination campaigns. It also saw a decrease in total approvals by the U.S. Food and Drug Administration (FDA). Nevertheless, there was no fall in the Biologics class, which was boosted through the authorization of 15 novel molecules, thus maintaining the figures achieved in previous years. Indeed, the decrease in approvals was only for the category of small molecules. Monoclonal antibodies (mAbs) continued to be the drug class with the most approvals, and cancer remained the most targeted disease, followed by autoimmune conditions, as in previous years. Interestingly, the FDA gave the green light to a remarkable number of bispecific Biologics (four), the highest number in recent years. Indeed, 2022 was another year without the approval of an antimicrobial Biologic, although important advancements were made in targeting new diseases, which are discussed herein. In this work, we only analyze the Biologics authorized in 2022. Furthermore, we also consider the orphan drugs authorized. We not only apply a quantitative analysis to this year’s harvest, but also compare the efficacy of the Biologics with those authorized in previous years. On the basis of their chemical structure, the Biologics addressed fall into the following classes: monoclonal antibodies; antibody-drug conjugates; and proteins/enzymes.

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“…In this context, the incentives provided by the FDA also drive greater resource allocation to these diseases. Annual growth in the development of Orphan Drugs is now expected [17,18].…”

Section: Orphan Drugsmentioning

confidence: 99%