MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN) has reached a significant milestone in the development of its gene-agnostic therapy, OCU400, as the European Commission has delivered a positive opinion designating it as an Advanced Therapy Medicinal Product (ATMP). The decision by the European Medicines Agency’s (EMA) Committee for Advanced Therapies (CAT) sets the stage for expedited regulatory review as Ocugen progresses toward global commercialization of the treatment by 2027.
OCU400 stands poised to become the first gene therapy to enter Phase 3 clinical trials with an indication for broad retinitis pigmentosa (RP), a rare genetic disease marked by severe vision loss and eventual blindness. With over 310,000 individuals in the U.S., Europe, and Canada affected by RP, OCU400 addresses critical unmet medical needs, especially for the 98% of patients suffering from forms of the disease for which no approved treatments exist.
“Receiving ATMP classification is another significant milestone toward bringing OCU400 to the market in Europe,” said Dr. Shankar Musunuri, Ocugen’s Chairman, CEO, and Co-founder. “This designation makes it possible to stay on track with our clinical and commercial strategy and potentially provide this novel modifier gene therapy candidate to all RP patients in the United States (U.S.) and Europe by 2027.”
Advancing a Groundbreaking Treatment for Retinitis Pigmentosa
The European Commission’s ATMP designation provides Ocugen with accelerated regulatory pathways and enhanced collaboration with the EMA. These advantages will support the company as it works toward completing its pivotal Phase 3 liMeliGhT trial and submitting its Marketing Authorization Application (MAA) in 2026.
The ongoing liMeliGhT trial aims to evaluate OCU400’s innovative approach to stabilizing and potentially improving retinal health across a broad range of RP genotypes. The trial includes 150 participants, randomly allocated across two genetic categories—those with RHO gene mutations and a gene-agnostic cohort—and treated with OCU400 or placed in an untreated control group.
“ We are encouraged by the EMA’s recognition of OCU400 as the Phase 3 liMeliGhT clinical trial advances,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “I look forward to working collaboratively with the EMA to address the unmet medical need that remains for nearly 98% of the RP patient population.”
A Novel Approach to Gene Therapy
Unlike traditional gene therapies that target specific mutations, OCU400 leverages its proprietary NR2E3 modifier gene platform to reset cellular pathways disrupted by RP. This gene-agnostic technique has demonstrated potential to restore cellular balance in the retina, providing hope for treating a broader spectrum of genetic variations associated with RP.
By regulating critical functions such as photoreceptor development, inflammation management, and cellular survival, OCU400 represents a transformational advancement in regenerative medicine for inherited retinal diseases.
Addressing Unmet Needs for RP Patients
RP is caused by mutations in more than 100 genes, leading to progressive vision impairment that currently has no broadly effective treatments. OCU400’s innovative approach positions it as a potentially life-changing therapy for a patient population that has long faced limited options.
With both the U.S. Food and Drug Administration (FDA) and the EMA agreeing that the liMeliGhT trial can serve as a single pivotal study for U.S. and European regulatory submissions, Ocugen anticipates filing for approvals in both regions upon completion of the Phase 3 study.
Looking Ahead
Ocugen’s achievement underscores its leadership in the field of gene therapy and its dedication to developing accessible solutions for underserved populations. Should OCU400 receive full regulatory approval, its potential to redefine RP care would mark a turning point not only for patients but also for the industry’s understanding of gene-agnostic therapies.
As the company advances its clinical and regulatory efforts, OCU400 stands as a promising symbol of hope for the RP community, offering the prospect of improved vision and quality of life for thousands of affected individuals. With ATMP classification secured, Ocugen is firmly on track to disrupt the landscape of inherited retinal disease treatment.
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