EMA grants orphan designation for Aldeyra’s eye treatment

Published 24/07/2025, 12:10
EMA grants orphan designation for Aldeyra’s eye treatment

LEXINGTON, Mass. - Aldeyra Therapeutics, Inc. (Nasdaq:ALDX), currently trading near $5.50 and showing strong momentum with a 10% gain over the past week according to InvestingPro data, announced Thursday that the European Medicines Agency (EMA) has granted Orphan Designation for its ADX-2191 (methotrexate intravitreal injection) for treating inherited retinal dystrophies of the rod-dominant phenotype, including retinitis pigmentosa.

Retinitis pigmentosa, which affects more than one million people worldwide, is a group of rare genetic eye diseases characterized by retinal cell death and progressive vision loss. Currently, there are no approved drug treatments for most forms of this condition. With a market capitalization of $331 million and strong analyst support, including three recent upward earnings revisions noted by InvestingPro, Aldeyra appears well-positioned to pursue this significant market opportunity.

This European designation complements the Orphan Drug Designation previously granted by the U.S. Food and Drug Administration for the same treatment. The EMA designation provides benefits including reduced regulatory fees, clinical protocol assistance, research grants, and up to 10 years of market exclusivity in the European Union. The company maintains a healthy financial position with a current ratio of 6.49 and more cash than debt on its balance sheet, providing runway for its development programs.

"Retinitis pigmentosa is a serious and incurable sight-threatening disease that represents a major unmet need in the field of ophthalmology," said Todd C. Brady, M.D., Ph.D., President and CEO of Aldeyra, in the press release. Investors tracking this development should note that Aldeyra’s next earnings report is scheduled for August 8, according to InvestingPro, which offers comprehensive financial analysis and 8 additional ProTips for the company.

The company reported that ADX-2191’s potential effectiveness is supported by Phase 2 clinical trial results announced in 2023, which showed improvements in retinal sensitivity following treatment. Aldeyra plans to initiate a Phase 2/3 clinical trial of ADX-2191 in retinitis pigmentosa in 2025.

ADX-2191 is a sterile, non-compounded intravitreal formulation of methotrexate designed to be vitreous-compatible and optimized for administration into the eye. Preclinical research has identified methotrexate’s activity in clearing misfolded rhodopsin, a visual cycle protein, suggesting potential benefit in treating genetic forms of retinitis pigmentosa characterized by this condition.

In other recent news, Aldeyra Therapeutics has made significant strides in its drug development efforts. The U.S. Food and Drug Administration has accepted Aldeyra’s resubmitted New Drug Application for reproxalap, a treatment for dry eye disease, with a target action date set for December 16, 2025. This follows a successful clinical trial that addressed previous methodological concerns highlighted by the FDA. Additionally, Aldeyra has received a Special Protocol Assessment Agreement from the FDA for its investigational drug ADX-2191, aimed at treating primary vitreoretinal lymphoma. This agreement outlines the design of a clinical trial comparing different dosages of the drug, which is expected to commence in the latter half of 2025. The trial will focus on cancer cell clearance in patients and is anticipated to provide results in 2026. These developments mark important milestones for Aldeyra as it progresses with its drug pipeline.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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