Ocugen’s Retinitis Pigmentosa Gene Therapy Available in EAP Ahead of BLA Submission
Participants are being dosed with OCU400 in the ongoing phase 3 liMeliGhT trial.
Lejla Vajzovic, MD, FASRS
Credit: Duke University
The FDA has approved an Expanded Access Program (EAP) for Ocugen’s OCU400 gene therapy program for adults with retinitis pigmentosa (RP).1
“Each clinical milestone achieved by OCU400 brings us closer to providing a potential one-time treatment for life to patients living with RP,” Shankar Musunuri, PhD, MBA, chairman, chief executive officer, and cofounder, Ocugen, said in a statement.1 “With positive Phase 1/2 study data and an ongoing Phase 3 liMeliGhT clinical trial, we now plan to work with clinicians, patients, and the RP community to provide access to OCU400 for eligible patients through our EAP. The EAP strengthens our commitment to serving RP patients—300,000 in the U.S. and Europe and 1.6 million globally.”
The EAP is available for adult patients in the US with early, intermediate to advanced RP with at least minimal retinal preservation who may benefit from the mechanism of action of OCU400 prior to approval of the Biologics License Application (BLA). This may include patients who participated in the OCU400 Phase 1/2 study and who qualify for dosing in the contralateral eye as well as patients who failed to meet inclusion criteria in the Phase 1/2 trial and ongoing Phase 3 liMeliGhT clinical trial (NCT06388200).
“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, Director, Duke Surgical Vitreoretinal Fellowship Program, Associate Professor of Ophthalmology with Tenure, Adult and Pediatric Vitreoretinal Surgery and Disease, Duke University Eye Center, and Retina Scientific Advisory Board Chair, Ocugen, added.1 “The OCU400 EAP gives RP patients access to this novel modifier gene therapy outside of the ongoing Phase 3 study.”
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Ocugen is actively dosing patients at least 8 years of age in the liMeliGhT clinical trial, starting with the first patient who was dosed in July 2024.2 The trial plans to enroll 150 participants, 75 with RHO gene mutations, and 75 participants who have mutations in other genes. Within each arm, participants will be randomized 2:1 to the treatment group (2.5 x1010 vg/eye of OCU400) and untreated control group, respectively. The trial will last 1 year, and its primary endpoint is achieving an improvement of at least 2 Lux levels from baseline in the study eyes on Luminance Dependent Navigation Assessment (LDNA). Other outcome measures include change in Low Luminance Visual Acuity, adverse events, and Patients Global Impression of Change score.
OCU400 is an adeno-associated virus (AAV) gene therapy delivering a nuclear hormone receptor gene called NR2E3. The therapy is designed to reset the dysfunctional gene network in patients with RP and reestablish a healthy cellular homeostasis via NR2E3’s regulation of photoreceptor development and maintenance, metabolism, phototransduction, inflammation, cell survival, and other physiological functions.
“We are pleased to make OCU400 available to patients beyond our Phase 3 liMeliGhT clinical trial through this EAP,” Huma Qamar, MD, MPH, CMI, Chief Medical Officer, Ocugen, added.1 "We are excited to expand our enrollment to include patients representing a diverse array of RP gene mutations. This program reflects our ongoing commitment to develop a safe and effective therapy for RP patients who may not have other treatment options.”
OCU400 has received orphan drug and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA. The European Medicines Agency (EMA) has accepted the US-based trial for submission of a Marketing Authorization Application (MAA) and Ocugen stated that OCU400 remains on track for targeted BLA and MAA approval in 2026.
