Key Takeaways

  • The FDA has granted Orphan Drug Designation to Octant’s OCT-980 for the treatment of retinitis pigmentosa (RP)
  • The designation includes rhodopsin-associated autosomal dominant RP, the indication for which OCT-980 is currently being developed
  • The investigational oral small molecule is currently being evaluated in a phase 1/2 clinical study

Octant, a clinical-stage drug discovery company developing correctors for protein misfolding diseases, announced that the FDA has granted Orphan Drug Designation to OCT-980 for the treatment of retinitis pigmentosa (RP). The designation includes rhodopsin-associated autosomal dominant RP (RHO-adRP), the indication for which OCT-980 is currently being developed.

According to the company, 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.

“People with RHO-adRP are living with progressive vision loss and no approved treatment options today,” said Sri Kosuri, cofounder and CEO of Octant, in a company press release. “This designation is another step forward as we advance OCT-980. With our phase 1b/2 study underway at US sites, we are focused on generating the clinical data needed to establish OCT-980 as an oral, disease-modifying option for RHO-adRP.”

RP is a group of inherited retinal disorders that cause progressive vision loss and can ultimately lead to blindness. RHO-adRP is caused by disease-associated variants in the RHO gene. Many of these variants reportedly result in misfolded and mistrafficked rhodopsin proteins that fail to localize properly within photoreceptor cells, contributing to progressive vision loss.

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 7 years of US market exclusivity following regulatory approval, subject to applicable requirements.

More information about the OCT-980 phase 1/2 clinical study is available on ClinicalTrials.gov (NCT07408232).