NR082, a gene therapy drug candidate for the treatment of Leber hereditary optic neuropathy, was granted orphan drug designation by the European Medicines Agency, according to a press release from Neurophth Therapeutics.
“The positive opinion from the [Committee for Orphan Medicinal Products] acknowledges the compelling IND-enabling data and clinical data of 186 subjects from three investigator-initiated trials,” Bin Li, MD, PhD, founder of Neurophth, said in the release.
In addition, the FDA recently cleared an investigational new drug application for NR082 for the treatment of