User fee goal dates in April include four novel agents with breakthrough therapy designations, including Pfizer’s hemophilia B gene therapy and Immunity Bio’s IL-15 superagonist complex for bladder cancer.

More than 100 biopharma executives and experts told Scrip their predictions for therapeutic area advances in the coming year. The recent commercial success of GLP-1s in diabetes and obesity and their potential in further disease areas fuelled excitement around the metabolic space. Expectations were also positive in neurology following the launch of Leqembi for Alzheimer’s disease in 2023, while the multiple opportunities to improve cancer treatment kept oncology top of the pile overall.

Safety signals were the most common cause of discontinued candidates in the breakthrough therapy designation program in 2023.

Neuroscience and oncology fuel high hopes for therapeutic R&D progress among 90-plus surveyed biopharma industry leaders.