Ocugen announces FDA approval of Expanded Access Program for patients with retinitis pigmentosa

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The United States Food and Drug Administration (FDA) notified Ocugen, Inc. to begin its expanded access program (EAP) for the treatment of patients, aged 18 and older, with retinitis pigmentosa (RP) with OCU400. This treatment is a modifier gene therapy product candidate currently seeing positive results in clinical trials.

Shankar Musunuri, PhD, MBA, Chairman, CEO, and Co-founder of Ocugen shared details about the status of OCU400, in the press release, saying, “Each clinical milestone achieved by OCU400 brings us closer to providing a potential one-time treatment for life to patients living with RP. With positive Phase 1/2 study data and an ongoing Phase 3 liMeliGhT (pronounced “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 US and Europe and 1.6 million globally.”

Generally, the EAP issuance 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.

In the case of the OCU400 EAP is available for patients 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). Ocugen is actively dosing patients in the Phase 3 liMeliGhT clinical trial.

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 of Ocugen noted that this candidate may be a long-term solution to this condition. In the press release, she said, “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. The OCU400 EAP gives RP patients access to this novel modifier gene therapy outside of the ongoing Phase 3 study.”

Similarly, Huma Qamar, Ocugen’s Chief Medical Officer reflected on the news and the current enrollment in clinical trials, saying, “We are pleased to make OCU400 available to patients beyond our Phase 3 liMeliGhT clinical trial through this EAP. 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.”

In previous releases, Ocugen noted that OCU400 has received orphan drug and Regenerative Medicine Advanced Therapy (RMAT) designations from FDA and that the European Medicines Agency (EMA) accepted the US-based trial for submission of a Marketing Authorization Application (MAA). With the dosing of patients in the Phase 3 clinical trial program underway, OCU400 remains on track for targeted BLA and MAA approval in 2026.

References:

1. Ocugen, Inc. Announces FDA Approval of Expanded Access Program for Patients with Retinitis Pigmentosa. Ocugen. August 5, 2024. Accessed August 5, 2024. https://www.biospace.com/ocugen-inc-announces-fda-approval-of-expanded-access-program-for-patients-with-retinitis-pigmentosa