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.
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. https://www.businesswire.com/news/home/20210804005122/en/Aldeyra-Therapeutics-Receives-Orphan-Drug-Designation-from-the-U.S.-Food-and-Drug-Administration-for-ADX-2191-to-Treat-Retinitis-Pigmentosa.