The European Medicines Agency (EMA) has formally recognized radiation maculopathy (RM) as a distinct clinical indication and granted orphan drug designation to Roca Therapeutics’s lead candidate, RCT002. This milestone represents the first regulatory acknowledgment of RM as more than a secondary complication, potentially paving the way for earlier diagnosis and therapeutic innovation.

Radiation maculopathy, a vision-threatening complication of ionizing radiation, is seen in patients treated for uveal melanoma and other ocular tumors, as well as head, neck, and brain cancers. Currently, no approved therapies exist, and off-label anti-VEGF treatment remains the default despite suboptimal outcomes. RCT002 is a first-in-class, small-molecule topical therapy designed to target both CXCR2 and DRAK1, with the aim of reducing resistant neovascularization, macrophage-driven inflammation, fibrosis, and oxidative stress.

Roca Therapeutics, a French biotechnology company, anticipates initiating first-in-human studies of RCT002 next year. After radiation maculopathy as its lead indication, the company intends to file additional applications exploring its effect in diabetic macular edema and other neovascular retinopathies. RP