Small molecule drugs for atopic dermatitis, rheumatoid arthritis, and hereditary angioedema.

Abstract

OBJECTIVE\nTo review recent trends in the development of targeted small molecule drugs (SMDs) for the treatment of immunologically driven disorders, including atopic dermatitis, rheumatoid arthritis, and hereditary angioedema.\n\n\nDATA SOURCES\nData sources include peer-reviewed published literature from the PubMed database, published abstracts from scientific/medical meetings, and medication information from the Drugs@FDA database.\n\n\nSTUDY SELECTIONS\nManuscripts with primary or retrospective trial results, manuscripts with patient or physician survey results, articles providing expert perspectives and commentary on chronic immunologic disorders, Food and Drug Administration (FDA) package inserts, and abstracts from scientific meetings were selected.\n\n\nRESULTS\nTargeted biologic therapies have greatly improved response rates and symptom relief for patients with chronic immunologically driven disorders over the last two decades. However, recent advances in the understanding of molecular pathways involved in the pathogenesis of these disorders has led to the development of novel targeted SMDs such as tofacitinib and berotralstat that can be delivered orally or topically. Few head-to-head studies that compare the safety and efficacy of biologics to SMDs in immunologically driven disorders exist, although some studies suggest that oral and topical modes of administration are preferred by patients and may improve patient quality of life (QoL) over time.\n\n\nCONCLUSION\nScientific advances have led to an increase in the development of targeted SMDs for the treatment of chronic immunologic disorders, which may revolutionize the management of these diseases. Head-to-head studies and real-world evidence are needed to fully compare treatment attributes between biologics and SMDs, including safety, efficacy, adherence, impact on QoL, and cost-effectiveness.