This is a rare and aggressive form of cancer for which there is no approved treatment currently.
Aldeyra president and CEO Todd Brady said: “Primary vitreoretinal lymphoma is a potentially fatal cancer that today is treated with off-label compounded formulations of methotrexate that are injected into the eye.
“The receipt of orphan designation from the EMA, in conjunction with the previously announced orphan drug Designation from the US Food and Drug Administration, is another important step forward for our retinal disease programme, complementing our ongoing clinical development of ADX-2191, a novel, vitreous-compatible formulation of methotrexate that is specifically designed for intraocular injection.”
A proposed trial aims to evaluate cancer cell clearance after 30 days of treatment in up to 20 subjects.
This clinical trial received a special protocol assessment agreement from the US Food and Drug Administration.
Participants will be randomly assigned in a 1:1 ratio to receive either a single or eight intraocular injections of ADX-2191.
The study is anticipated to commence in the latter half of 2025 and conclude in 2026.
The EMA grants the designation to biologics and drugs designed to treat rare, life-threatening, or chronically debilitating conditions that impact fewer than five in every 10,000 individuals within the European Union.
This designation offers various incentives, such as reduced regulatory fees, assistance with clinical protocols, research grants, and up to ten years of market exclusivity.