Ocugen, Inc. has received the green light from the FDA to launch an expanded access program (EAP) for adult patients diagnosed with retinitis pigmentosa (RP) and treated with OCU400, the company’s lead investigational asset.
First things first: Let’s get familiar with OCU400.
As a modifier gene therapy candidate based on NR2E3—a nuclear hormone receptor (NHR) gene—OCU400 is intended to regulate physiological functions within the retina.
- How it will do this: By delivering an adeno-associated viral (AAV) vector via subretinal injection.
And its intended effect: Ideally, to reset the affected/altered gene cell networks and reestablish retinal homeostasis—potentially stabilizing cells and rescuing compromised photoreceptors from degenerative processes.
Walk me through its regulatory journey so far.
In December 2022, OCU400 was granted Orphan Drug designation by the FDA for both RP associated with RHO mutations and Leber Congenital Amaurosis (LCA).
- Then in December 2023, the gene therapy was granted Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA.
More recently: In April 2024, the FDA approved OCU400’s investigational new drug (IND) amendment.
- The significance of this: The amendment enabled Ocugen to initiate a multicenter, assessor-blinded, randomized phase 3 liMeliGhT trial (NCT06388200), with the first patient dosing confirmed in June 2024.
And the bigger picture?
With the recent patient dosing and overall trial initiation, OCU400 became the first gene therapy candidate to enter into the phase 3 clinical level with a broad RP indication.
Alrighty, now talk about EAP.
This FDA program is intended to provide a potential pathway for patients diagnosed with serious or immediately life-threatening diseases/conditions (such as RP) to gain access to an investigational medical product (such as OCU400) outside of clinical trials when no comparable or adequate alternative therapy options exist.
- Qualifying medical products for EAP have not been FDA-cleared or approved by the FDA, or yet deemed to be safe and effective for their specific use
And in the case of OCU400?
Ocugen’s EAP is available for “patients with early, intermediate-to-advanced RP with at least minimal retinal preservation who may benefit” from OCU400’s mechanism of action (MOA), according to the company.
Note: This access will be available for RP patients up until a future Biologics License Application (BLA) for OCU400 is approved by the FDA.
- And no—with the phase 3 liMEliGhT study ongoing—a BLA has not yet been submitted.
Speaking of that trial …
Ocugen also emphasized that patient enrollment is ongoing, noting that the study has expanded enrollment to include “patients representing a diverse array of RP gene mutations,” stated Lejla Vajzovic, MD, FASRS, Ocugen Scientific Advisory Board chair.
Lastly, what should we keep an eye out for from the company?
Keep in mind that a phase 1/2 study on OCU400 is still ongoing, with data expected around its expected December 2024 conclusion.
Plus, according to Clinical Trials, the liMEliGhT study is slated to conclude in October 2026 … so interim results will likely be released before then.
And as for any future BLA plans: The company stated it is on track for their targeted 2026 submission.