Aldeyra Therapeutics' ADX-2191 Receives Orphan Status for Rare Lymphoma Treatment

Aldeyra Therapeutics has received orphan drug designation from the European Medicines Agency (EMA) for ADX-2191, a treatment for rare and aggressive primary large B-cell lymphomas in immune-privileged sites, including primary vitreoretinal lymphoma. The company's focus on developing treatments for rare and underserved conditions positions it uniquely within the competitive biotechnology landscape. Despite its promising pipeline, Aldeyra currently reports no revenue, and the company faces operational challenges with a negative return on equity (ROE) and return on assets (ROA).

Lexington, Mass., August 28, 2025 – Aldeyra Therapeutics (Nasdaq: ALDX) has announced 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. This designation, which is intended for rare, life-threatening, or chronically debilitating diseases affecting fewer than five in 10,000 people in the European Union, provides Aldeyra with certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants, and up to 10 years of market exclusivity.

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 median survival for newly diagnosed patients is less than five years, and no approved treatments are currently available. The current standard of care involves off-label compounded formulations of methotrexate injected into the eye.

ADX-2191, a sterile, non-compounded intravitreal formulation of methotrexate, is designed to be vitreous-compatible and optimized for excipient composition, viscosity, density, tonicity, pH, concentration, and volume of administration. The drug has previously received FDA Orphan Drug Designation for the treatment of primary vitreoretinal lymphoma and retinitis pigmentosa, and EMA Orphan Designation for the treatment of inherited retinal dystrophies of the rod-dominant phenotype, including retinitis pigmentosa.

A proposed clinical trial, which received Special Protocol Assessment agreement from the U.S. Food and Drug Administration, 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 trial is expected to begin in the second half of 2025 and conclude in 2026.

While Aldeyra's focus on developing treatments for rare and underserved conditions positions it uniquely within the competitive biotechnology landscape, the company currently reports no revenue and faces operational challenges with a negative return on equity (ROE) and return on assets (ROA). The company's ability to successfully navigate these challenges and bring ADX-2191 to market will be crucial for its long-term success.

References:
[1] https://finance.yahoo.com/news/aldeyra-therapeutics-receives-orphan-designation-110100779.html