Top Selling (2026) Small Molecule Orphan Drugs: A Journey into Their Chemistry-Reference-Cited by-同舟云学术

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

Published:2023-01-04 Issue:2 Volume:24 Page:930
ISSN:1422-0067
Container-title:International Journal of Molecular Sciences
language:en
Short-container-title:IJMS

Author:

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

Abstract

This review describes, from a chemical point of view, the top “blockbuster” small molecule orphan drugs according to their forecasted sales in 2026. Orphan drugs are intended for the treatment, prevention, or diagnosis of a rare disease or condition. These molecules are mostly addressed to the treatment of rare forms of cancer. The respiratory and central nervous systems represent other common therapeutic subcategories. This work will show how the orphan drugs market has significantly grown and will account for a consistent part of prescriptions by 2026.

Publisher

MDPI AG

Subject

Inorganic Chemistry,Organic Chemistry,Physical and Theoretical Chemistry,Computer Science Applications,Spectroscopy,Molecular Biology,General Medicine,Catalysis

Link

https://www.mdpi.com/1422-0067/24/2/930/pdf

Reference76 articles.

1. Milne, C.-P., and Cabanilla, L.A. (2007). Comprehensive Medicinal Chemistry II, Elsevier.

2. (2022, October 26). Designating an Orphan Product: Drugs and Biological Products, Available online: https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/designating-orphan-product-drugs-and-biological-products.

3. (2022, October 26). EMA Orphan Designation: Overview. Available online: https://www.ema.europa.eu/en/human-regulatory/overview/orphan-designation-overview.

4. (2022, October 26). The Story behind the Orphan Drug Act, Available online: https://www.fda.gov/industry/fdas-rare-disease-day/story-behind-orphan-drug-act.

5. Rare Diseases and Orphan Drugs;Aronson;Br. J. Clin. Pharmacol.,2006

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