Octant Wins FDA Orphan Drug Status for Retinitis Pigmentosa Drug

Octant has received Orphan Drug Designation from the US Food and Drug Administration (FDA) for OCT-980, an investigational oral drug being developed to treat retinitis pigmentosa, a group of inherited eye diseases that can cause progressive vision loss and blindness.

Announced on October 27, 2026, the designation covers retinitis pigmentosa (RP), including rhodopsin-associated autosomal dominant retinitis pigmentosa (RHO-adRP). Octant is currently evaluating OCT-980 in a Phase 1/2 clinical trial in the United States.

Retinitis pigmentosa affects the retina, the light-sensitive tissue at the back of the eye. Over time, damage to the retina can make it harder for people to see in low light and can gradually reduce their peripheral vision. In some cases, the disease can lead to severe vision loss.

RHO-adRP is caused by changes in the rhodopsin (RHO) gene. These changes can cause rhodopsin proteins to fold incorrectly or fail to reach the right location within retinal cells. This can damage the light-sensing cells and contribute to progressive vision loss.

OCT-980 is a small-molecule drug designed to address this problem at its source. It aims to correct protein misfolding and help rhodopsin move to the correct location within cells. Unlike treatments that focus only on managing symptoms, Octant is investigating whether this approach could help address an underlying cause of the disease.

The drug is taken orally and remains experimental. Its safety and effectiveness have not yet been established, and further clinical research will be needed to determine whether it can slow or prevent vision loss in patients.

The FDA’s Orphan Drug Designation is intended to encourage the development of treatments for rare diseases affecting fewer than 200,000 people in the United States. The designation can provide benefits such as tax credits for eligible clinical trials, exemptions from certain FDA application fees and the possibility of seven years of market exclusivity after approval, subject to regulatory requirements.

Octant said the designation is another step in its efforts to develop a potential treatment for people living with RHO-adRP, for whom approved treatment options remain limited.

The ongoing Phase 1/2 study will help researchers assess OCT-980’s safety and gather early evidence of its potential benefit. The results will be important in deciding whether the drug can advance to later-stage clinical trials.

The development highlights growing research into genetic and protein-related causes of inherited eye diseases. If successful, OCT-980 could offer a new approach to treating patients with rhodopsin-related retinitis pigmentosa. However, its potential benefits will depend on the outcome of clinical testing.