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Aldeyra Therapeutics Receives Orphan Designation from the European Medicines Agency for ADX-2191 for the Treatment of Inherited Retinal Dystrophies including Retinitis Pigmentosa
LEXINGTON, Mass., July 24, 2025--Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra) today announced that the European Medicines Agency (EMA) has granted Orphan Designation for ADX-2191 (methotrexate intravitreal injection) for the treatment of inherited retinal dystrophies of the rod-dominant phenotype, including retinitis pigmentosa. There are currently no approved drug treatments for patients with most forms of retinitis pigmentosa, a clinical group of rare genetic eye diseases characterized
About this update from Aldeyra Therapeutics, Inc.
[{"type":"text","content":"LEXINGTON, Mass., July 24, 2025--(BUSINESS WIRE)--Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra) today announced that the European Medicines Agency (EMA) has granted Orphan Designation for ADX-2191 (methotrexate intravitreal injection) for the treatment of inherited retinal dystrophies of the rod-dominant phenotype, including retinitis pigmentosa. There are currently no approved drug treatments for patients with most forms of retinitis pigmentosa, a clinical group of rare genetic eye diseases characterized by retinal cell death and loss of vision. Retinitis pigmentosa affects more than one million people worldwide and remains a significant cause of inherited blindness.","length":682,"tagName":"p"},{"type":"text","content":""Retinitis pigmentosa is a serious and incurable sight-threatening disease that represents a major unmet need in the field of ophthalmology," stated Todd C. Brady, M.D., Ph.D., President and CEO of Aldeyra. "The receipt of Orphan Designation from the EMA, in conjunction with the previously announced Orphan Drug Designation from the U.S. Food and Drug Administration, highlights the importance of developing a treatment option for patients suffering from retinitis pigmentosa."","length":498,"tagName":"p"},{"type":"text","content":"The potential activity of ADX-2191 in retinitis pigmentosa is supported by results from a Phase 2 clinical trial, announced in 2023, which demonstrated improvements from baseline in retinal sensitivity following treatment. A planned Phase 2/3 clinical trial of ADX-2191 in retinitis pigmentosa is expected to initiate in 2025.","length":326,"tagName":"p"},{"type":"text","content":"The EMA grants orphan designation to drugs and biologics intended for the treatment, diagnosis, or prevention of rare, life-threatening, or chronically debilitating diseases or conditions that affect fewer than five in 10,000 people in the European Union. Orphan designation allows companies certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants, and up to 10 years of market exclusivity in the European Union.","length":455,"tagName":"p"},{"type":"text","content":"About ADX-2191","length":14,"tagName":"p"},{"type":"text","content":"ADX-2191 (methotrexate intravitreal injection) is a sterile, non-compounded intravitreal formulation of methotrexate...