A decade of approved first-in-class small molecule orphan drugs: Achievements, challenges and perspectives.
Eur J Med Chem
; 243: 114742, 2022 Dec 05.
Article
em En
| MEDLINE
| ID: mdl-36155354
ABSTRACT
In the past decade (2011-2020), there was a growing interest in the discovery and development of orphan drugs for the treatment of rare diseases. However, rare diseases only account for a population of 0.65-1 which usually occur with previously unknown biological mechanisms and lack of specific therapeutics, thus to increase the demands for the first-in-class (FIC) drugs with new biological targets or mechanisms. Considering the achievements in the past 10 years, a total of 410 drugs were approved by U.S. Food and Drug Administration (FDA), which contained 151 FIC drugs and 184 orphan drugs, contributing to make up significant numbers of the approvals. Notably, more than 50% of FIC drugs are developed as orphan drugs and some of them have already been milestones in drug development. In this review, we aim to discuss the FIC small molecules for the development of orphan drugs case by case and highlight the R&D strategy with novel targets and scientific breakthroughs.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Produção de Droga sem Interesse Comercial
/
Doenças Raras
Limite:
Humans
País/Região como assunto:
America do norte
Idioma:
En
Ano de publicação:
2022
Tipo de documento:
Article