The U.S. Food and Drug Administration has approved an Expanded Access Program for OCU400, a novel modifier gene therapy candidate for the treatment of adults with retinitis pigmentosa.

The Expanded Access Program (EAP) “allows patients who have unmet medical needs with serious or life-threatening conditions to access treatments outside of a clinical trial that are not yet approved by the FDA,” according to press release information. 

OCU400 treatment is based on the nuclear hormone receptor gene NR2E3, which regulates crucial physiological functions within the retina, such as photoreceptor development, metabolism, phototransduction, inflammation, and cell survival. The therapy aims to reset the dysfunctional gene network in retinal cells of retinitis pigmentosa (RP) patients to restore healthy cellular homeostasis and potentially improve vision.

Patients aged 18 and older with early, intermediate, or advanced RP who have at least minimal retinal preservation now have access to OCU400 through the EAP. The program also includes patients who participated in the OCU400 Phase 1/2 study and qualify for dosing in the contralateral eye, as well as those who did not meet inclusion criteria for the Phase 1/2 and ongoing Phase 3 liMeliGhT trials but could benefit.

“Each clinical milestone achieved by OCU400 brings us closer to providing a potential one-time treatment for life to patients living with RP,” said Dr. Shankar Musunuri in the press release.

 “RP patients with mutations in multiple genes currently have no therapeutic options. As a retinal surgeon, I am encouraged by the therapeutic potential of OCU400 to provide long-term benefit,” Lejla Vajzovic, MD, FASRS, added.