The Food and Drug Administration has granted Rare Pediatric Disease designation to HORA-PDE6b, an investigational gene therapy developed by EyeDNA Therapeutics for retinitis pigmentosa caused by PDE6b gene mutations.

Symptoms of PDE6b-related retinitis pigmentosa often appear in childhood and can lead to blindness by midlife. There are no approved treatments for this condition, which affects up to 3,000 people in the United States. HORA-PDE6buses an AAV5-based vector to deliver a functional PDE6b gene to photoreceptor cells in the retina, aiming to halt or delay vision loss.

Early results from a phase 1/2 trial (NCT03328130) indicate a strong safety profile and meaningful improvements in visual function after 24 months of treatment. The Rare Pediatric Disease designation makes the therapy eligible for a priority review voucher, which can be sold or used to accelerate FDA review of another drug.

EyeDNA, a subsidiary of French genetic medicine company Coave Therapeutics, reported in a press release that it anticipates seeking FDA approval for HORA-PDE6b by 2028. The release said that the company is also exploring accelerated approval pathways in the US and Europe for this treatment. RP