The FDA has granted orphan drug designation to Mitotech for Visomitin, a topical treatment for Leber hereditary optic neuropathy, according to a press release.
“The U.S. orphan drug designation is highly encouraging for our efforts to bring Visomitin to young people whose lives have been radically affected by LHON,” Natalia Perekhvatova, CEO of Mitotech, said in the release. “Visomitin has an outstanding safety profile, and our preliminary human studies have demonstrated effectiveness in LHON.”
In a phase 2a study, Visomitin (SkQ1) showed consistent visual acuity