Octant announced in a press release that the US Food and Drug Administration (FDA) has granted orphan drug designation to OCT-980 for the treatment of retinitis pigmentosa. The designation includes rhodopsin-associated autosomal dominant retinitis pigmentosa (RHO-adRP), the indication for which OCT-980 is currently being developed.

OCT-980 is an investigational oral small molecule designed to address RHO-adRP at its source by correcting the underlying protein misfolding and restoring proper cellular trafficking of rhodopsin. It is currently being evaluated in a phase 1/2 clinical study.

The FDA grants orphan drug designation to drugs intended to treat rare diseases or conditions. The designation provides certain development incentives, including potential tax credits for qualified clinical trials, exemption from certain FDA application fees, and eligibility for seven years of US market exclusivity following regulatory approval, subject to applicable requirements. RP