August 6, 2024

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

Ocugen, Inc. is working on a new potential treatment for retinitis pigmentosa (RP), an eye disease that causes vision loss over time. Their gene therapy, OCU400, is a possible one-time treatment being studied in the liMeliGhT trial. After promising results in early studies (Phase 1/2), they are now in Phase 3, the last step before the company can apply for approval from the U.S. Food and Drug Administration (FDA).

Usually, people can try new treatments only by joining a clinical trial or waiting until the treatment is fully approved, which can take years. The liMeliGhT trial was initially designed only for people with RP caused by a change in the RHO gene. Through the Expanded Access Program (EAP), the FDA is letting Ocugen give OCU400 to up to 75 people with RP, regardless of their genetic changes. To qualify, patients must still have some healthy photoreceptors left, even if they are in the advanced stages of RP.

What this means for Usher syndrome: Since RP is part of Usher syndrome, this program means all individuals 18 and older can be considered for the OCU400 gene therapy as long as they meet the necessary conditions.

For more information, visit the clinical trials webpage.

Disclaimer: The information contained on the Usher Syndrome Coalition website and other online properties is provided for your information only. The Coalition is neither a medical service nor a scientific organization. It provides information concerning the various types of Usher syndrome that includes information developed or derived from unrelated third parties over which the Coalition has no involvement or control. The Usher Syndrome Coalition does not independently, nor in conjunction with others, attest to, deny, or comment upon the merits of the accuracy of the information provided by unrelated third parties. No reliance on medical or scientific information contained on this website should be taken by any person. You should always consult with and be guided by your Physician’s advice when considering treatment based on research results.