FDA approves expanded access program for retinitis pigmentosa gene therapy candidate
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The FDA approved Ocugen’s expanded access program for the treatment of retinitis pigmentosa with OCU400, according to a press release.
“One major challenge with RP is that it can be caused by mutations in any of over 100 different genes, but most therapies focus on addressing just one gene,” Huma Qamar, MD, MPH, CMI, chief medical officer of Ocugen, told Healio. “OCU400 expresses a master regulator gene called NR2E3 that helps reestablish homeostasis within the retinal cell environment regardless of the underlying mutation, which will address an unmet need for over 98% of RP patients.”
The expanded access program (EAP) will allow patients aged 18 years and older with early and intermediate to advanced retinitis pigmentosa (RP) with at least minimal retinal preservation to be treated with the modifier gene therapy product candidate outside of clinical trials and before the approval of its biologics license application.
OCU400 is currently being investigated in the phase 3 liMeliGhT clinical trial.
As Healio previously reported, OCU400 was granted orphan drug designation as well as regenerative medicine advanced therapy designation. Ocugen said the therapy is “on track” for approval of its biologics license and marketing authorization applications in 2026.
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