NEW YORK – Ocugen on Monday said it will provide its gene therapy candidate OCU400 to adult patients with retinitis pigmentosa through an expanded access program (EAP).
The US Food and Drug Administration has approved the Malvern, Pennsylvania-based biotech's EAP, within which OCU400 will be available to certain patients with early intermediate-to-advanced retinitis pigmentosa who have at least minimal retinal preservation and could benefit from the gene therapy.
Before the FDA approved the EAP, patients could only access OCU400 within a clinical trial. Ocugen has evaluated OCU400 in a Phase I/II study and is assessing it in the ongoing Phase III liMeliGhT trial. Patients may be eligible to receive the gene therapy through the EAP if they participated in the Phase I/II study and qualify for dosing in the non-treated eye or if they were ineligible for that study or for liMeliGhT.
Retinitis pigmentosa is a group of eye disorders caused by mutations in more than 60 genes, most commonly occurring in the RHO gene. OCU400 aims to treat the condition by "resetting" the dysfunctional gene network in retinitis pigmentosa patients and reestablish healthy cellular homeostasis by delivering a copy of the NR2E3 gene that regulates physiological functions within the retina.
"RP patients with mutations in multiple genes currently have no therapeutic options," Lejla Vajzovic, an associate professor of ophthalmology at the Duke University Eye Center and chair of Ocugen's retina scientific advisory board, said in a statement.
OCU400 has orphan drug and regenerative medicine advanced therapy designations from the FDA. Ocugen has said it is on track to submit regulatory applications for the gene therapy in the US and EU in 2026.