A European recognition for a neglected disease: Roca Therapeutics has obtained from the European Medicines Agency (EMA) the orphan drug designation for its drug candidate RCT002. This status marks the official recognition of radiation maculopathy as a disease in its own right, after having long been considered only a complication. This advancement enables reinforced regulatory support and accelerates market access.
An innovative solution for a disease without treatment: RCT002 is a “first-in-class” eye drop that targets several mechanisms involved in radiation maculopathy: inflammation, neovascularization, fibrosis and oxidative stress. It offers a non-invasive alternative where current options are off-label and poorly effective. Its entry into clinical phase is planned for 2026, a structuring milestone for the biotech.
A key stage for a Riviera startup supported locally: Founded in 2021 in Nice, Roca Therapeutics is backed by a strong network of public and private actors from the South region. This success strengthens its positioning as a leading player in innovative ophthalmology. The company is currently preparing a fundraising round to finance its clinical trial.
In its press release of August 26, 2025, the biotech details the announcement:
Roca Therapeutics (Roca), a pioneer in non-invasive, first-in-class therapies for severe ocular diseases, today announces the granting of Orphan Drug Designation (ODD) by the European Medicines Agency (EMA) for its lead clinical candidate, RCT002, in the treatment of Radiation Maculopathy. 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 recognized 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 ionizing radiation. It commonly affects patients who have undergone radiotherapy, not only for ocular or periocular tumors, 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 two years from its proprietary platform, RCT002 is a gamechanging first-in-class small molecule eye-drop. RCT002 has been rationally designed to safely and concomitantly address resistant neovascularization, 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 specialized treatments for patients experiencing vision loss after radiotherapy for eye, head and neck or brain cancers,” said Zaki Sellam, executive chairman 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, on track to commence in 2026.
Roca has secured early-stage financial and strategic backing from the 3B Future Health Fund. The company is also supported by a broad coalition of institutional partners, including SATT Sud-Est, Bpifrance, Université Côte d’Azur, Eurobiomed, Incubateur PACA-Est, Métropole Nice Côte d’Azur, the Provence-Alpes-Côte d’Azur Regional Council and European Regional Development Fund (FEDER). These strategic partnerships and financial milestones reflect strong confidence in RCT002’s potential to reshape the therapeutic landscape in retinal diseases and position Roca as a frontrunner in ophthalmic innovation.
