LEXINGTON, Mass.– Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) announced today that the European Medicines Agency (EMA) has granted Orphan Designation for ADX-2191 (methotrexate intravitreal injection, USP) for the treatment of primary large B-cell lymphomas of immune-privileged sites, including primary vitreoretinal lymphoma. There are currently no approved therapies for primary vitreoretinal lymphoma, a rare and aggressive cancer that affects an estimated 100 to 200 people annually in the European Union.
“Primary vitreoretinal lymphoma is a potentially fatal cancer that today is treated with off-label compounded formulations of methotrexate injected directly into the eye,” said Todd C. Brady, M.D., Ph.D., President and CEO of Aldeyra. “The receipt of Orphan Designation from the EMA, alongside our Orphan Drug Designation from the U.S. FDA, represents another important step forward for our retinal disease program. It complements our ongoing clinical development of ADX-2191, a vitreous-compatible formulation of methotrexate specifically designed for intraocular injection.”
A planned clinical trial, which has received Special Protocol Assessment agreement from the U.S. FDA, is expected to enroll up to 20 patients in a randomized 1:1 study comparing cancer cell clearance after 30 days of therapy with either a single intraocular injection or eight injections of ADX-2191. Previous studies suggest that the frequency of methotrexate injections is linked to cancer cell clearance in primary vitreoretinal lymphoma, with an average of about five injections often required to achieve clearance. The trial is expected to begin in the second half of 2025 and conclude in 2026.
The EMA grants orphan designation to drugs and biologics intended for rare, life-threatening, or chronically debilitating diseases affecting fewer than five in 10,000 people in the EU. The designation provides sponsors with benefits such as reduced regulatory fees, clinical protocol assistance, research grants, and up to 10 years of market exclusivity.
