Release Date: 20-Sep-2021
US FDA has given orphan drug designation for intravitreal injection (ADX-2191) for the treatment of retinitis pigmentosa (RP). As of now, no approved drugs are available for the treatment of this rare illness. RP is characterized by retinal cell death and vision loss. It is likely to affect 82,000 to 110,000 people in the U. S and about 1 in 4,000 people in the whole world. Methotrexate is an inhibitor of dihydroflic reductase, which is a protein involved in the cellular activation and replication of tissues.
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.