The Food and Drug Administration (FDA) has granted Orphan Drug designation to ADX-2191 (methotrexate for intravitreal injection) for the treatment of retinitis pigmentosa.

Retinitis pigmentosa is a rare, inherited degenerative eye disease caused by a breakdown and loss of cells in the retina resulting in cell death and loss of vision. ADX-2191 is an intravitreal formulation of methotrexate that inhibits dihydrofolate reductase, an enzyme involved in cellular replication and activation.

“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, MD, PhD, President and CEO of Aldeyra. “ADX-2191 has now received orphan designations for 3 distinct clinical indications, highlighting the broad platform potential of ADX-2191 to treat an array of rare retinal disorders.”

The Agency also granted Orphan Drug designation to ADX-2191 for the treatment of primary vitreoretinal lymphoma, along with Fast Track and Orphan Drug designations for the prevention of proliferative vitreoretinopathy.

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Aldeyra Therapeutics receives Orphan Drug designation from the U.S. Food and Drug Administration for ADX-2191 to treat retinitis pigmentosa. News release. Aldeyra Therapeutics, Inc. Accessed August 4, 2021. 

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