Ocugen, Inc. Announces Positive Scientific Advice from the European Medicines Agency Related to the Approval Pathway for OCU410ST鈥擬odifier Gene Therapy for Stargardt Disease
Ocugen (NASDAQ: OCGN) received positive scientific advice from the European Medicines Agency (EMA) regarding the approval pathway for OCU410ST, its gene therapy treatment for Stargardt disease. The EMA's Committee for Medicinal Products for Human Use (CHMP) has accepted a single U.S.-based trial for Marketing Authorization Application (MAA) submission.
The Phase 2/3 GARDian3 clinical trial will enroll 51 participants, with 34 receiving OCU410ST treatment and 17 serving as controls. The therapy has shown promising Phase 1 results, including 48% slower lesion growth and a statistically significant 2-line/9-letter gain in visual acuity (p=0.031). The company plans to complete enrollment by Q1 2026 and submit a Biologics License Application (BLA) in H1 2027.
The treatment targets approximately 100,000 Stargardt patients in the U.S. and Europe combined, who currently have no approved treatment options.Ocugen (NASDAQ: OCGN) ha ricevuto un parere scientifico positivo dall'Agenzia europea per i medicinali (EMA) sul percorso di autorizzazione per OCU410ST, la sua terapia genica per la malattia di Stargardt. Il Comitato per i medicinali per uso umano (CHMP) dell'EMA ha accettato uno studio unico basato negli Stati Uniti per la presentazione della domanda di autorizzazione all'immissione in commercio (MAA).
Lo studio clinico di fase 2/3 GARDian3 arruoler脿 51 partecipanti, di cui 34 riceveranno il trattamento OCU410ST e 17 fungeranno da controllo. La terapia ha mostrato risultati promettenti nella fase 1, inclusa una crescita delle lesioni pi霉 lenta del 48% e un guadagno statisticamente significativo di 2 linee/9 lettere nell'acuit脿 visiva (p=0.031). L'azienda prevede di completare l'arruolamento entro il primo trimestre del 2026 e di presentare una Biologics License Application (BLA) nella prima met脿 del 2027.
Il trattamento 猫 rivolto a circa 100.000 pazienti con Stargardt negli Stati Uniti e in Europa, che attualmente non dispongono di opzioni terapeutiche approvate.
Ocugen (NASDAQ: OCGN) recibi贸 asesoramiento cient铆fico positivo por parte de la Agencia Europea de Medicamentos (EMA) sobre la v铆a de aprobaci贸n de OCU410ST, su terapia g茅nica para la enfermedad de Stargardt. El Comit茅 de Medicamentos de Uso Humano (CHMP) de la EMA acept贸 un 煤nico ensayo basado en Estados Unidos para la presentaci贸n de la solicitud de autorizaci贸n de comercializaci贸n (MAA).
El ensayo cl铆nico de fase 2/3 GARDian3 reclutar谩 a 51 participantes, de los cuales 34 recibir谩n OCU410ST y 17 actuar谩n como controles. La terapia mostr贸 resultados prometedores en la fase 1, incluyendo un crecimiento de las lesiones un 48% m谩s lento y una ganancia estad铆sticamente significativa de 2 l铆neas/9 letras en la agudeza visual (p=0.031). La compa帽铆a planea completar el reclutamiento para el primer trimestre de 2026 y presentar una Biologics License Application (BLA) en la primera mitad de 2027.
El tratamiento est谩 dirigido a aproximadamente 100.000 pacientes con Stargardt en EE.聽UU. y Europa, que actualmente no disponen de opciones terap茅uticas aprobadas.
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Ocugen (NASDAQ: OCGN) a re莽u un avis scientifique positif de l'Agence europ茅enne des m茅dicaments (EMA) concernant la voie d'autorisation de OCU410ST, son traitement par th茅rapie g茅nique contre la maladie de Stargardt. Le Comit茅 des m茅dicaments 脿 usage humain (CHMP) de l'EMA a accept茅 un essai unique men茅 aux 脡tats-Unis pour la soumission de la demande d'autorisation de mise sur le march茅 (MAA).
L'essai clinique de phase 2/3 GARDian3 recrutera 51 participants, dont 34 recevront OCU410ST et 17 serviront de t茅moins. Le traitement a montr茅 des r茅sultats prometteurs en phase 1, notamment une croissance des l茅sions 48% plus lente et un gain statistiquement significatif de 2 lignes/9 lettres d'acuit茅 visuelle (p=0.031). La soci茅t茅 pr茅voit de terminer le recrutement d'ici le T1 2026 et de d茅poser une Biologics License Application (BLA) au premier semestre 2027.
Le traitement cible environ 100 000 patients atteints de Stargardt aux 脡tats-Unis et en Europe, qui n'ont actuellement aucune option th茅rapeutique approuv茅e.
Ocugen (NASDAQ: OCGN) erhielt von der Europ盲ischen Arzneimittelagentur (EMA) positive wissenschaftliche Beratung zum Zulassungsweg f眉r OCU410ST, seine Gentherapie gegen die Stargardt-Krankheit. Der Ausschuss f眉r Humanarzneimittel (CHMP) der EMA hat eine einzige in den USA durchgef眉hrte Studie f眉r die Einreichung des Zulassungsantrags (MAA) akzeptiert.
Die Phase-2/3-Studie GARDian3 wird 51 Teilnehmer einschlie脽en, von denen 34 OCU410ST erhalten und 17 als Kontrollen dienen. Die Therapie zeigte vielversprechende Ergebnisse in Phase 1, darunter ein um 48% verlangsamtes L盲sionswachstum und einen statistisch signifikanten Zuwachs von 2 Linien/9 Buchstaben in der Sehsch盲rfe (p=0.031). Das Unternehmen plant, die Rekrutierung bis Q1 2026 abzuschlie脽en und im ersten Halbjahr 2027 eine Biologics License Application (BLA) einzureichen.
Die Behandlung richtet sich an sch盲tzungsweise 100.000 Patienten mit Stargardt in den USA und Europa, f眉r die derzeit keine zugelassenen Behandlungsoptionen bestehen.
- Phase 1 trial showed 48% slower lesion growth and significant 2-line/9-letter visual acuity improvement
- EMA acceptance of single U.S.-based trial will reduce time and costs for EU approval
- Treatment addresses large market of 100,000 Stargardt patients with no current treatment options
- Clear timeline for BLA filing in first half of 2027
- Recent regulatory achievements including Rare Pediatric Disease Designation and IND clearance
- Extended timeline to potential approval with BLA filing not expected until H1 2027
- Relatively small trial size of 51 participants may limit statistical power
Insights
Ocugen's OCU410ST therapy shows promising progress with EMA accepting a single pivotal trial for Stargardt disease approval, significantly accelerating EU market pathway.
Ocugen has secured a significant regulatory advancement for OCU410ST, their modifier gene therapy for Stargardt disease. The European Medicines Agency (EMA) has reviewed and accepted the company's Phase 2/3 GARDian3 clinical trial design as potentially sufficient for a Marketing Authorization Application, without requiring additional European trials. This streamlined regulatory pathway could substantially reduce time-to-market in Europe.
The EMA's confidence appears rooted in compelling Phase 1 data showing
The ongoing Phase 2/3 trial employs a sophisticated adaptive design with 51 participants, including an interim analysis of 24 subjects at 8 months. The primary endpoint focuses on reducing atrophic lesion size, with visual acuity measures as key secondary endpoints. This design balances scientific rigor with accelerated development timelines.
For Stargardt disease patients - approximately 100,000 in the US and Europe combined - this therapy represents potential hope where no approved treatments currently exist. The one-time subretinal injection approach could offer a transformative treatment modality.
With the recent Rare Pediatric Disease Designation, IND clearance, first patient dosing, and now this EMA feedback, Ocugen is maintaining momentum toward their projected BLA filing in 2027. The company's ambitious goal of three BLAs in three years demonstrates their strategic commitment to advancing multiple candidates through their pipeline simultaneously.
MALVERN, Pa., Aug. 13, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) reviewed the study design, endpoints and planned statistical analysis of the ongoing pivotal confirmatory OCU410ST Phase 2/3 GARDian3 clinical trial for Stargardt disease and provided acceptability of a single U.S.-based trial for submission of a Marketing Authorization Application (MAA).
EMA provided this opinion based on safety and tolerability that OCU410ST demonstrated in the Phase 1 GARDian trial, including
鈥淭his positive opinion endorses a single trial as the basis for both BLA and MAA submissions and brings us closer to providing a one-time, modifier gene therapy to approximately 100,000 Stargardt patients in the U.S. and Europe combined,鈥� said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. 鈥淲e are very encouraged about the prospect of addressing the unmet medical need that exists for these patients who currently have no approved treatment options available to them.鈥�
The EMA opinion is an extremely favorable outcome, as it will potentially reduce the time and cost to gain marketing authorization in the EU. Alignment with the EMA follows recent important milestones for the OCU410ST program, including Rare Pediatric Disease Designation (RPDD) in May, IND clearance in June, and first patient dosing in July. With enrollment scheduled to be complete in the first quarter of 2026 the Company remains on track for a BLA filing in the first half of 2027, aligned with its goal of three BLAs in the next three years.
About OCU410ST
OCU410ST utilizes an AAV delivery platform for the retinal delivery of the聽RORA聽(RAR-Related Orphan Receptor A) gene. It represents Ocugen鈥檚 modifier gene therapy approach, which is based on Nuclear Hormone Receptor (NHR)聽RORA that regulates pathophysiological pathways linked to Stargardt disease, such as lipofuscin formation, oxidative stress, complement formation, inflammation, and cell survival networks.
About Stargardt Disease
Stargardt disease is a genetic eye disorder that causes retinal degeneration and vision loss. Stargardt disease is the most common form of inherited macular degeneration. The progressive vision loss associated with Stargardt disease is caused by the degeneration of photoreceptor cells in the central portion of the retina called the macula.
Decreased central vision due to loss of photoreceptors in the macula is the hallmark of Stargardt disease. Some peripheral vision is usually preserved. Stargardt disease typically develops during childhood or adolescence, but the age of onset and rate of progression can vary. The retinal pigment epithelium (RPE), a layer of cells supporting photoreceptors, is also affected in people with Stargardt disease.
About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene therapies to address major blindness diseases and offer hope for patients across the globe. We are making an impact on patient鈥檚 lives through courageous innovation鈥攆orging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to address significant unmet medical need for large patient populations through our gene-agnostic approach. Discover more at聽聽and follow us on聽听补苍诲听.
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 鈥減redicts,鈥� 鈥渂elieves,鈥� 鈥減otential,鈥� 鈥減roposed,鈥� 鈥渃ontinue,鈥� 鈥渆stimates,鈥� 鈥渁nticipates,鈥� 鈥渆xpects,鈥� 鈥減lans,鈥� 鈥渋ntends,鈥� 鈥渕ay,鈥� 鈥渃ould,鈥� 鈥渕ight,鈥� 鈥渨ill,鈥� 鈥渟hould,鈥� 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 OCU410ST 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 more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled 鈥淩isk Factors鈥� in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.
Contact:
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FAQ
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