MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN), a Pennsylvania-based biopharmaceutical company, has scored a significant regulatory milestone this week with the U.S. Food and Drug Administration (FDA) granting Regenerative Medicine Advanced Therapy (RMAT) designation to its investigational product, OCU400, for treating retinitis pigmentosa (RP) associated with RHO mutations.
The FDA’s RMAT designation is a notable validation of OCU400’s potential as a game-changing gene therapy approach, according to Arun Upadhyay, PhD, Chief Scientific Officer and Head of R&D at Ocugen. The decision underscores the urgency to bring a therapeutic option to RP patients who face the risk of blindness.
The RMAT designation, part of the 21st Century Cures Act, aims to facilitate the development and review of regenerative medicine therapies intended to treat, modify, reverse, or cure serious conditions. The designation offers sponsor companies benefits similar to the fast track and breakthrough therapy programs, including early interactions with the FDA. Ocugen is currently working with the FDA to finalize the Phase 3 protocol necessary to advance the clinical development of OCU400.
Current data suggests that OCU400, which employs Ocugen’s modifier gene therapy approach, could provide treatment benefits to a broader group of RP and Leber congenital amaurosis (LCA) patients. This gene-agnostic mechanism of action would be a significant boon to those suffering from these vision-threatening disorders. Ocugen plans to submit additional efficacy and safety data for OCU400 in RP and LCA patients to potentially expand the RMAT designation to a wider patient population.
RHO mutations affect more than 10,000 of the approximately 110,000 people in the U.S. diagnosed with RP. A recent Phase 1/2 trial update revealed that 86% of RHO mutation subjects experienced either stabilization or improvement in their Multi-Luminance Mobility Test (MLMT) scores, with 29% demonstrating a 3 Lux luminance level improvement. To date, there are no treatment options available for RP patients with RHO gene mutations.
The RMAT designation could potentially fast-track OCU400 to the market, offering a new lifeline to RP and LCA patients. For investors, this development underscores Ocugen’s commitment to innovative solutions for unmet medical needs and could enhance the company’s standing in the biopharmaceutical sector.
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