Orphan Disease

ADVM-062, a novel gene therapy candidate, received orphan drug designation from the FDA for the treatment of blue cone monochromacy, according to a press release from Adverum Biotechnologies.

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 FDA has granted orphan drug designation to SIG-001, an investigational therapy under evaluation for hemophilia A, according to a press release from the drug’s manufacturer.