ADX-2191 Wins FDAs Orphan Drug Designation

Release Date: 15-Sep-2021

Aldeyra Therapeutics earns Orphan Drug Designation from US FDA for ADX-2191 (methotrexate for intravitreal injection) for the treatment of primary vitreoretinal lymphoma (PVRL). ADX-2191 inhibits dihydrofolate reductase, an enzyme involved in cellular replication and activation. Methotrexate is the most generally used intravitreal medication for the treatment of PVRL.


Previously, ADX-2191 was granted orphan drug status and fast track designation by the FDA for the prevention of proliferative vitreoretinopathy which is a rare but serious sight-threatening retinal disease with no approved treatment. There are no approved treatments for PVRL that is an aggressive, rare, high-grade cancer that affects about 2,800 people in the U.S, with around 600 new cases diagnosed annually.


The FDA's orphan-drug program gives special status to drugs and biologics for diseases and disorders that affect fewer than 200,000 people in the U.S and it also gives several benefits, like tax credits for qualified clinical trials, potential seven years of market exclusivity after approval and exemption from user fees.

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