This regulatory milestone is the result of Roca’s strategic and sustained efforts to elevate radiation maculopathy from being viewed merely as a secondary medical complication to being formally recognised by the EMA as a distinct and differentiated clinical indication.
Radiation maculopathy is a vision-threatening retinal complication that arises from exposure of the macular region to ionising radiation.
It commonly affects patients who have undergone radiotherapy, not only for ocular or periocular tumours, but also for malignancies of the head and neck or brain, where incidental radiation to the eye may occur.
For this indication, there is currently no approved standard of care and off-label treatment remains the prevailing clinical practice, albeit with poor results.
Developed within 2 years from its proprietary platform, RCT002 is a game-changing first-in-class small molecule eye-drop.
RCT002 has been rationally designed to safely and concomitantly address resistant neovascularisation, inflammation, fibrosis and oxidative stress.
“Recognition of radiation maculopathy as a distinct indication marks a critical step forward in patient care — enabling earlier diagnosis, targeted therapeutic development and expanded access to specialised treatments for patients experiencing vision loss after radiotherapy for eye, head and neck or brain cancers,” said Zaki Sellam, Executive Chair of Roca Therapeutics.
“This orphan designation also opens the door to accelerated innovation and regulatory support, creating a clear pathway for investment in a high-need under-addressed therapeutic area.”
Roca is currently fundraising to support RCT002’s first-in-human clinical trial in 2026.