EMA grants orphan drug designation for Roca’s treatment of radiation maculopathy
Ella Day | August 28, 2025 | News story | Medical Communications, Research and Development | European Medicines Agency (EMA), Oncology, Opthalmology, Roca Therapeutics, maculopathy
The European Medicines Agency (EMA) has granted Orphan Drug Designation to Roca Therapeutics for its lead candidate RCT002, marking the first time radiation maculopathy has been formally recognised as a distinct medical indication.
Radiation maculopathy is a rare, vision-threatening condition that can develop after radiotherapy for cancers such as uveal melanoma, head and neck tumours or brain malignancies. It occurs when radiation damages the macula – the part of the retina responsible for central vision. Currently, there is no approved treatment and patients are provided with only off-label therapies which often yield poor results.
RCT002 is an eye drop designed to address several drivers of disease progression, including abnormal blood vessel growth, inflammation and oxidative stress. If successful, it would be the first non-invasive therapy for patients at risk of vision loss after radiotherapy.
“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,” said Zaki Sellam, executive chairman of Roca Therapeutics. “This orphan designation also opens the door to accelerated innovation and regulatory support.”
Roca plans to begin first-in-human clinical trials of RCT002 in 2026 and is currently raising funds to support the programme.
Ella Day
28/8/25
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