MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN), a biopharmaceutical company, recently announced that it has received alignment from the U.S. Food and Drug Administration (FDA) on the design of a pivotal Phase 3 clinical trial. The trial aims to assess the safety and efficacy of OCU400, a treatment for patients with RHO and other gene mutations associated with Retinitis Pigmentosa (RP).
The news is a significant step towards Ocugen’s goal to bring first-in-class, gene-agnostic therapies to market and make them accessible to patients worldwide. As stated by Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen, the company plans to initiate the Phase 3 clinical trial in early 2024.
The FDA’s agreement on the Phase 3 study design followed a multidisciplinary meeting and was based on preliminary results from an ongoing Phase 1/2 study. Key aspects of the Phase 3 trial—including the study endpoint, patient enrollment strategy, and a one-year study duration—received the regulatory body’s approval.
The Phase 3 trial will enroll a wider group of RP patients, including those with the most common RHO gene mutation. This broad inclusion is based on the potentially gene-agnostic mechanism of action of OCU400.
With orphan drug and Regenerative Medicine Advanced Therapy (RMAT) designations already granted for OCU400, the FDA’s alignment on the Phase 3 study design bolsters Ocugen’s position as it pursues product development and licensure for OCU400.
Retinitis Pigmentosa affects approximately 110,000 patients in the United States and 1.6 million patients globally. Over 10% of these patients have the RHO genetic mutation. The advancement of OCU400 to Phase 3 clinical development marks a crucial step in addressing the unmet needs in the RP patient community.
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