Release Date: 25-Sep-2021
US FDA has granted orphan drug designation to RLF-100 (aviptadil) for the treatment of Sarcoidosis. Sarcoidosis is a rare disease in which the inflammatory process includes the alveoli (air sacs), small bronchi, and small blood vessels. As the disease growths, small lumps, or granulomas, appear in the affected tissues that tend to remain inflamed and become scarred (fibrotic). Granulomas are structured masses composed of activated immune cells (macrophages, lymphocytes, mast cells and fibroblasts.
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.
Many patients who are suffering with sarcoidois are left with permanent lung damage as they undergo a chronic course where problems such as pulmonary fibrosis are common and irreversible. As of now in U.S, there are about 140,000 sarcoidosis patients.