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Scientific advances, in combination with government incentives and commercial opportunity, have fuelled strong investment in orphan drugs, resulting in many innovative therapies. Here, we discuss the approach of the FDA to a range of issues that remain crucial to maintaining this momentum, such as the use of the totality of evidence in evaluating orphan drugs.
CARB-X, a public–private partnership aimed at bolstering the antibiotic pipeline, funded a diverse set of 17 early-stage drug development projects in its first year.
Nora Volkow, director of the National Institute on Drug Abuse (NIDA), discusses her ambitious aim of halving the time needed to develop non-opioid analgesics and anti-addiction drugs.
This analysis of US drug launches from the past decade indicates that commercial success has become more difficult to achieve, owing to factors such as increased competition, greater challenges in achieving therapeutic differentiation and payer pressures.
New therapies for idiopathic pulmonary fibrosis (IPF) are urgently needed. Here, Moraet al. discuss our current understanding of the mechanisms driving IPF, highlighting the parallels between fibrosis and ageing. Potential avenues for drug discovery and development are described, including progress made and current obstacles.
WD40 repeat (WDR) domain-containing proteins are involved in numerous protein complexes that have been linked to disease. Schapira and colleagues describe recent advances in targeting WDR domains with small molecules to potently inhibit protein–protein interactions, and discuss the potential for the diversity and druggability of WDR domains to open up new pathways for therapeutic intervention.
G protein-coupled receptors (GPCRs) are key drug targets. In this article, Marshall and colleagues discuss the progress made towards generating GPCR-targeting antibodies, including which antigen formats and antibody platforms have been most successful. They review the current pipeline and outline outstanding challenges in antibody generation.