FDA Grants Orphan Drug Designation To ADX-2191 (Intravitreal Methotrexate)

Release Date: 17-Sep-2021

Aldeyra Therapeutics receives Orphan Drug Designation from US FDA for ADX-2191 to treat retinitis pigmentosa. ADX-2191 is an intravitreal formulation of methotrexate that inhibits dihydrofolate reductase, an enzyme involved in cellular replication and activation. ADX-2191 can also be used to treat primary vitreoretinal lymphoma, along with Fast Track and Orphan Drug designations for the prevention of proliferative vitreoretinopathy. Retinitis pigmentosa is a rare, inherited degenerative eye disease caused by a breakdown and loss of cells in the retina causing loss of vision and in cell death.


The FDA’s Orphan Drug Program provides financial incentives to companies developing drugs for rare diseases and conditions. Through the program, eligible drugs can be granted tax credits and other benefits.

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