Expanded Access Policy

EXPANDED ACCESS PROGRAM

Mitigating circumstances and situations may exist when enrollment in a clinical trial is not possible (patient ineligibility) or feasible (distance to a trial is too far), that may preclude an individual with a serious condition or life-threatening illness from access to an investigational drug product (IDPs). In cases where an individual is deemed ineligible to participate in a clinical trial, an expanded access program (EAP) presents another option for patients with serious medical conditions and/or life-threatening disease to gain access to IDPs outside of participating in a clinical trial. If both the patient and the patient’s treating physician believe that there is potential benefit(s) that could be derived from the IDP and there is no comparable or satisfactory alternative treatment available to manage that condition, the patient’s treating physician may assist the patient in gaining access to the EAP. IDPs available through EAP do not make claims or assertions that the IDP is safe or efficacious to diagnose, treat or monitor the patient’s serious medical conditions. And while an EAP is not considered a clinical trial, the program must operate within certain parameters because scientific data will be collected. In addition to FDA review of the EAP, an Investigational Review Board (IRB) should review the expanded access protocol and informed consent to ensure the patient is informed about the nature of the treatment.

ABOUT THE OCU400 EAP

The OCU400 EAP is a U.S.-only protocol for (1) eligible adult RP patients, 18 years and older, with early, intermediate to advanced disease with at least minimal retinal preservation, (2) patients who participated in the OCU400 Phase 1/2 study and who qualify for dosing in the contralateral eye, (3) patients who failed to meet inclusion criteria in the Phase 1/2 trial and ongoing Phase 3 liMeliGhT clinical trial who could benefit from OCU400, and (4) RP patients who can benefit from the mechanism of action of OCU400 prior to BLA approval.

Additional information on the OCU400 EAP will be available on https://clinicaltrials.gov/study/NCT06574997

To learn more, please contact:

Mahvish Tafseer, MD
Phone: +1 (484)-224-3409
The code to access the voicemail is 1357
It is named OCU400 EAP
Email: eap@ocugen.com

NOTE: Ocugen may require up to 5-7 business days to acknowledge receipt of the written request and will provide a written response to all written requests in a timely manner. Certain factors may affect the length of the review period and may include but are not limited to obtaining permission from regulatory authorities, the need for additional information from the requestor, or other factors beyond Ocugen’s control. Ocugen recognizes that the decision to approve or deny a written request is complex and will assist where possible to make the process smooth and seamless. Ocugen commits to: (i) conduct a clinical review of all written requests by an appropriate medical officer, (ii) decide each written request in an equitable and fair manner, and (iii) use the available scientific and clinical evidence in making each decision.