MP-004, our lead candidate for Retinitis Pigmentosa, is granted Orphan Drug Designation by the FDA
At Miramoon Pharma, we are delighted to announce that MP-004, our lead product in development for the treatment of retinitis pigmentosa, has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA).
MP-004 is an investigational drug with a dual mechanism of action designed to prevent calcium mishandling from “leaky” RyR channels by interacting with FKBP12 and reducing the formation of highly reactive deleterious species. Through this dual mechanism of action, MP products are intended to keep the intracellular concentration of calcium below the toxic levels that usually trigger cell death, particularly in highly excitable cells which are vulnerable to imbalances of the intracellular calcium content.
Orphan Drug Designation (ODD) is bestowed by the FDA upon products designed for diagnosing, treating, or preventing rare diseases and disorders impacting fewer than 200,000 individuals in the United States. ODD offers companies various incentives aimed at fostering the development of promising therapeutic drugs and addressing unmet medical needs.