With no approved therapies globally for Stargardt's, competition in the indication is increasing in Japan, where Kubota Vision is seeking for conditional approval for emixustat, while Belite Bio’s contender tinlarebant has received Sakigake designation as a local Phase III program continues.

Six new breakthrough therapy designations have been announced, all for novel agents, but approvals were fewer and limited to new indications and formulations.

Incyte’s Jakafi-dominated portfolio gained Opzelura as a second source of significant revenue in 2021. While further diversification has been limited, new and existing R&D assets may deliver upside in 2024.

Immuno-oncology will remain a major force at both FDA’s drugs and biologics centers, but watch for psychiatry and antibiotics to make some noise; rare pediatric diseases will remain prominent as the priority review voucher program heads into the sunset.