1. Oct 05, 2020

FDA grants orphan drug designation to retinitis pigmentosa treatment

The U.S. Food and Drug Administration has granted orphan drug designation for EYS611 (Eyevensys) for the treatment of retinitis pigmentosa (RP), according to a company press release.

EYS611 is a DNA plasmid that encodes for the human transferrin protein.

“We are delighted to have received orphan-drug designation from the FDA as it is an important regulatory milestone. We look forward to translating our unique non-viral gene therapy program to patients with RP, to slow the progression of this degenerative retinal disease with no currently approved treatment that compromises patients’ vision and eventually lead to blindness,” said Thierry Bordet, PhD, Chief Scientific Officer, in a company news release.

Data from preclinical testing that demonstrates that EYS611 is safe and effective for preserving photoreceptors and retina functionality in acute toxicity and inherited rat models of retinal degeneration was published in the journal Pharmaceutics in September 2020.

Read the full press release here.