Ocugen receives FDA alignment on key points of Phase 3 clinical trial design for OCU 400
During a multidisciplinary meeting with FDA, based on preliminary results from an ongoing Phase 1/2 study, the company received alignment on key points of the Phase 3 study design.
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Ocugen Inc announced it received alignment from the FDA on key aspects of the Phase 3 clinical trial design to assess the safety and efficacy of OCU400 in patients with RHO and other gene mutations associated with Retinitis Pigmentosa (RP).
“This news brings us even closer to fulfilling our mission to bring our first-in-class, gene-agnostic therapies to market and provide access to patients globally,” Shankar Musunuri, PhD, MBA, chairman, CEO, and co-founder of Ocugen, said in a news release. “We look forward to beginning the Phase 3 clinical trial, which we plan to initiate in early 2024.”
According to the company, during a multidisciplinary meeting with FDA, based on preliminary results from an ongoing Phase 1/2 study, it received alignment on key points of the Phase 3 study design—including the study endpoint, patient enrollment strategy, and study duration of 1 year.
Moreover, according to the news release, the Phase 3 clinical trial will enroll a broader group of RP patients, including patients with the most common RHO gene mutation, based on OCU400’s potentially gene-agnostic mechanism of action.1
The company also noted OCU400 already has orphan drug and RMAT designations in place, and the FDA’s alignment on key aspects of the Phase 3 study design positions it to move forward in pursuing product development and licensure for OCU400.
Furthermore, the company also noted in its news release there are about 110,000 patients in the United States with RP and 1.6 million patients globally. Of these patients, more than 10% have the RHO genetic mutation. Advancing OCU400 to Phase 3 clinical development will be an important step toward addressing unmet needs in the RP patient community.1
Reference:
Ocugen gains FDA alignment on key aspects of OCU400-modifier gene therapy-pivotal phase 3 study design. Ocugen, Inc. Accessed December 21, 2023. https://ir.ocugen.com/news-releases/news-release-details/ocugen-gains-fda-alignment-key-aspects-ocu400-modifier-gene.
