Aldeyra Receives Orphan Designation for ADX-2191 in EU for Rare Lymphoma Treatment.

Aldeyra (ALDX) received orphan designation from the European Medicines Agency for ADX-2191 to treat primary large B-Cell lymphomas of immune-privileged sites, including primary vitreoretinal lymphoma. There is currently no approved treatment for this rare, aggressive cancer that affects approximately 100-200 people per year in the EU.

Lexington, Mass., August 28, 2025 — Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) has received Orphan Designation from the European Medicines Agency (EMA) for ADX-2191, a novel intravitreal formulation of methotrexate, to treat primary large B-Cell lymphomas of immune-privileged sites, including primary vitreoretinal lymphoma [1]. This designation is a significant milestone for Aldeyra, as it highlights the potential of ADX-2191 to address a currently unmet medical need.

Primary vitreoretinal lymphoma is a rare, aggressive, and potentially fatal retinal cancer that affects approximately 100 to 200 people per year in the European Union. The lack of approved treatments for this condition underscores the importance of Aldeyra's development efforts. The EMA's Orphan Designation provides certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants, and up to 10 years of market exclusivity in the European Union [2].

ADX-2191 is designed to be vitreous-compatible and optimized for excipient composition, viscosity, density, tonicity, pH, concentration, and volume of administration. The drug is intended to be used for specific rare retinal diseases, including primary vitreoretinal lymphoma and retinitis pigmentosa. The company has previously received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for ADX-2191 to treat primary vitreoretinal lymphoma and retinitis pigmentosa [1].

A proposed clinical trial, which received Special Protocol Assessment agreement from the FDA, is designed to compare cancer cell clearance after 30 days of therapy in up to 20 patients following 1:1 randomization to receive either a single intraocular injection or eight intraocular injections of ADX-2191. The frequency of methotrexate injections has been linked to cancer cell clearance in patients with primary vitreoretinal lymphoma, with approximately five injections on average observed to achieve cancer cell clearance [1].

The clinical trial is expected to begin in the second half of 2025 and conclude in 2026. The receipt of Orphan Designation from the EMA, in conjunction with the previously announced Orphan Drug Designation from the FDA, is a significant step forward for Aldeyra's retinal disease program, complementing the company's ongoing clinical development of ADX-2191.

References:

[1] https://www.marketscreener.com/news/aldeyra-therapeutics-inc-receives-orphan-designation-from-the-european-medicines-agency-for-adx-21-ce7c50dfd18df521
[2] https://finance.yahoo.com/news/aldeyra-therapeutics-receives-orphan-designation-110100779.html