Ocugen, Inc. Receives Positive Opinion for OCU400 Advanced Therapy Medicinal Product Classification from European Commission

Ocugen received ATMP classification for OCU400, advancing its Phase 3 trial for retinitis pigmentosa gene therapy in Europe.

Quiver AI Summary

Ocugen, Inc. has announced that the European Commission has given a positive opinion for the classification of its gene therapy product, OCU400, as an Advanced Therapy Medicinal Product (ATMP) by the European Medicines Agency’s Committee for Advanced Therapies. OCU400 is the first gene therapy to reach Phase 3 trials for retinitis pigmentosa (RP), a disease affecting approximately 310,000 patients across the U.S., EU, and Canada. This classification is a significant milestone as it facilitates a faster regulatory review process and allows Ocugen to interact closely with EMA during development. Ocugen plans to file for marketing authorization in both the U.S. and Europe following the completion of the Phase 3 trial, which is currently enrolling participants. The therapy aims to address unmet medical needs in the RP patient population, which has no current approved treatments to halt disease progression.

Potential Positives

• Ocugen received a positive opinion from the European Medicines Agency (EMA) Committee for Advanced Therapies (CAT) for the OCU400 Advanced Therapy Medicinal Product (ATMP) classification, marking a significant milestone in the development process.
• OCU400 is the first gene therapy to reach Phase 3 for a broad retinitis pigmentosa indication, potentially benefiting a large patient population.
• The ATMP classification accelerates the regulatory review timeline, allowing Ocugen to potentially provide the gene therapy to patients in the U.S. and Europe by 2027.
• Ocugen plans to file for regulatory approval simultaneously in the U.S. and Europe, reflecting a strong commitment to addressing the unmet medical need in the retinitis pigmentosa patient community.

Potential Negatives

• The press release emphasizes the regulatory uncertainties by detailing the risks associated with the company's forward-looking statements, which may indicate potential challenges in achieving their clinical and commercial timelines.
• The company is still in the early stages of a pivotal Phase 3 trial with a limited sample size of 150 participants, indicating potential difficulties in drawing conclusive results or meeting regulatory expectations.
• The press statement points out that there are no approved treatment options for retinitis pigmentosa, highlighting the high stakes and pressure on Ocugen to deliver results successfully.

FAQ

What is OCU400 and its significance?

OCU400 is a gene therapy for retinitis pigmentosa (RP) and the first to enter Phase 3 with a broad indication.

What does ATMP classification mean for OCU400?

The ATMP classification accelerates the regulatory review process and ensures closer collaboration with the EMA.

What is the sample size for the Phase 3 liMeliGhT trial?

The Phase 3 liMeliGhT clinical trial has a sample size of 150 participants, split into two arms.

How does OCU400 address the needs of RP patients?

OCU400 is designed to be gene-agnostic, potentially treating multiple forms of RP associated with over 100 gene mutations.

When does Ocugen plan to file for marketing authorization?

Ocugen intends to file for Marketing Authorization Application in both the U.S. and Europe after the Phase 3 trial is completed in 2026.

Disclaimer: This is an AI-generated summary of a press release distributed by GlobeNewswire. The model used to summarize this release may make mistakes. See the full release here.

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Full Release

MALVERN, Pa., Feb. 03, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that the European Commission has provided a positive opinion from the European Medicines Agency’s (EMA) Committee for Advanced Therapies (CAT) for OCU400 Advanced Therapy Medicinal Product (ATMP) classification. OCU400 is the first gene therapy to enter Phase 3 with a broad retinitis pigmentosa (RP) indication.

“Receiving ATMP classification is another significant milestone toward bringing OCU400 to the market in Europe,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “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.”

ATMP classification is granted to medicines that can offer groundbreaking opportunities for the treatment of disease and accelerates the regulatory review timeline of this potential one-time gene therapy for life. Additionally, this classification allows Ocugen to interact with EMA more frequently for scientific advice and protocol assistance as the Company pursues Marketing Authorization Application (MAA) filing in 2026.

Underscoring the vital need for gene-agnostic treatments for diseases with multiple mutations such as RP, both the U.S. Food and Drug Administration (FDA) and EMA have acknowledged that the ongoing single, pivotal Phase 3 trial of OCU400 can suffice for Biologics License Application (BLA)/MAA submissions. Ocugen intends to file simultaneously in the U.S. and Europe upon completion of the Phase 3 trial.

The Phase 3 OCU400 liMeliGhT clinical trial is currently enrolling. The study has a sample size of 150 participants—one arm of 75 participants with

RHO

gene mutations and the other arm with 75 participants that are gene agnostic. In each arm, participants will be randomized 2:1 to the treatment group (2.5 x 10

¹⁰

vg/eye of OCU400) and untreated control group, respectively. Patients eight years of age and older, with early through late-stage advancement of RP, are being recruited to participate in the liMeliGhT study.

“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.”

RP affects nearly 310,000 patients in the U.S., EU, and Canada. Currently, RP is associated with mutations in more than 100 genes and there are no approved treatment options that slow or stop the progression of multiple forms of RP.

OCU400 is the Company’s gene-agnostic modifier gene therapy product based on NHR gene,

NR2E3

.

NR2E3

regulates diverse physiological functions within the retina—such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation and cell survival networks. Through its drive functionality, OCU400 resets altered/affected cellular gene networks and establishes homeostasis—a state of balance, which has the potential to improve retinal health and function in patients with inherited retinal diseases.

About Ocugen, Inc.



Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at

www.ocugen.com

and follow us on

X

and

LinkedIn

.

Cautionary Note on Forward-Looking Statements

This


press


release


contains


forward-looking


statements


within


the


meaning


of


The


Private


Securities


Litigation


Reform


Act


of


1995,


including,


but


not limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines,


which


are


subject


to


risks


and


uncertainties.


We


may,


in


some


cases,


use


terms


such


as


“predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,”


or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including,


but


not


limited


to,


the


risks


that


preliminary,


interim


and


top-line


clinical


trial


results


may


not


be


indicative


of,


and


may


differ


from,


final


clinical data;


the ability of OCU400 to perform in humans in a manner consistent with nonclinical, preclinical or previous clinical study data;


that


unfavorable


new


clinical


trial


data


may


emerge


in


ongoing


clinical


trials


or


through


further


analyses


of


existing


clinical


trial


data;


that


earlier non-clinical


and


clinical


data


and


testing


of


may


not


be


predictive


of


the


results


or


success


of


later


clinical


trials;


and


that


that


clinical


trial


data


are subject to differing interpretations and assessments, including by regulatory authorities.


These


and


other


risks


and


uncertainties


are


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fully described


in


our


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filings


with


the


Securities


and


Exchange


Commission


(SEC),


including


the


risk


factors


described


in


the


section


entitled


“Risk Factors”


in


the


quarterly


and


annual


reports


that


we


file


with


the


SEC.


Any


forward-looking


statements


that


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make


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release


speak


only


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date


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Contact:



Tiffany Hamilton


AVP, Head of Communications



Tiffany.Hamilton@ocugen.com

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.