Ocugen Announces European Medicines Agency Grants Orphan Medicinal Product Designation for Modifier Gene Therapy Candidate OCU410ST for Treatment of ABCA4-Associated Retinopathies including Stargardt Disease
Ocugen announced that the European Medicines Agency (EMA) has granted orphan medicinal product designation for OCU410ST to treat ABCA4-associated retinopathies including Stargardt disease. The designation offers benefits including protocol assistance, reduced fees, research grants, and 10 years of market exclusivity. Phase 1 dosing of the Phase 1/2 OCU410ST GARDian trial is complete, with the DSMB recommending progression to Phase 2. Preliminary data showed an 84% reduction in atrophic lesion growth in treated eyes versus untreated eyes. The treatment targets approximately 100,000 patients in the U.S. and Europe combined. OCU410ST previously received FDA orphan drug designation in April 2023.
Ocugen ha annunciato che l'Agenzia Europea per i Medicinali (EMA) ha concesso la designazione di farmaco orfano per OCU410ST, destinato a trattare le retinopatie associate ad ABCA4, inclusa la malattia di Stargardt. Questa designazione offre vantaggi come assistenza per il protocollo, tariffe ridotte, sovvenzioni per la ricerca e 10 anni di esclusività sul mercato. La somministrazione della Fase 1 della sperimentazione GARDian di OCU410ST è completata, con il DSMB che raccomanda il passaggio alla Fase 2. I dati preliminari hanno mostrato una riduzione dell'84% nella crescita delle lesioni atrofizzate negli occhi trattati rispetto a quelli non trattati. Il trattamento mira a circa 100.000 pazienti negli Stati Uniti e in Europa insieme. OCU410ST ha precedentemente ricevuto la designazione di farmaco orfano dalla FDA nell'aprile 2023.
Ocugen anunció que la Agencia Europea de Medicamentos (EMA) ha concedido la designación de producto medicinal huérfano para OCU410ST para tratar las retinopatías asociadas a ABCA4, incluida la enfermedad de Stargardt. Esta designación ofrece beneficios como asistencia en el protocolo, tarifas reducidas, subvenciones para la investigación y 10 años de exclusividad en el mercado. La dosificación de la Fase 1 del ensayo GARDian de OCU410ST está completa, con el DSMB recomendando la progresión a la Fase 2. Los datos preliminares mostraron una reducción del 84% en el crecimiento de lesiones atróficas en los ojos tratados frente a los no tratados. El tratamiento se dirige a aproximadamente 100,000 pacientes en EE. UU. y Europa combinados. OCU410ST recibió anteriormente la designación de medicamento huérfano de la FDA en abril de 2023.
Ocugen은 유럽 의약청(EMA)가 ABCA4 관련 망막병을 치료하기 위해 OCU410ST에 대한 희귀 의약품 지정을 부여했다고 발표했습니다. 이 지명은 프로토콜 지원, 수수료 인하, 연구 보조금 및 10년의 시장 독점권 등 혜택을 제공합니다. OCU410ST GARDian 시험의 1상 투약이 완료되었으며, DSMB는 2상으로의 진행을 권장했습니다. 예비 데이터에 따르면 치료한 눈에서 위축 병변 성장의 84% 감소가 나타났습니다. 이 치료는 미국과 유럽의 약 100,000명의 환자를 대상으로 하고 있습니다. OCU410ST는 2023년 4월 FDA의 희귀 약물 지정을 이미 받았습니다.
Ocugen a annoncé que L'Agence Européenne des Médicaments (EMA) a accordé la désignation de médicament orphelin pour OCU410ST afin de traiter les rétinopathies associées à ABCA4, y compris la maladie de Stargardt. Cette désignation offre des avantages tels que l'assistance au protocole, des frais réduits, des subventions à la recherche et 10 ans d'exclusivité sur le marché. La posologie de la phase 1 de l'essai GARDian de OCU410ST est terminée, le DSMB recommandant une progression vers la phase 2. Les données préliminaires ont montré une réduction de 84 % de la croissance des lésions atrophiques dans les yeux traités par rapport aux yeux non traités. Le traitement vise environ 100 000 patients aux États-Unis et en Europe. OCU410ST a précédemment reçu la désignation de médicament orphelin de la FDA en avril 2023.
Ocugen hat angekündigt, dass die Europäische Arzneimittel-Agentur (EMA) die Orphan-Medicine-Produktbezeichnung für OCU410ST zur Behandlung von ABCA4-assoziierten Retinopathien, einschließlich der Stargardt-Krankheit, erteilt hat. Diese Bezeichnung bietet Vorteile wie Protokollhilfe, reduzierte Gebühren, Forschungsstipendien und 10 Jahre Marktexklusivität. Die Dosisverabreichung der Phase 1 der OCU410ST GARDian-Studie ist abgeschlossen, wobei der DSMB die Fortschreitung zur Phase 2 empfiehlt. Erste Daten zeigten eine 84%ige Reduzierung des Wachstums atrophischer Läsionen in behandelten Augen im Vergleich zu unbehandelten Augen. Die Behandlung zielt auf etwa 100.000 Patienten in den USA und Europa ab. OCU410ST erhielt zuvor im April 2023 die Orphan-Drug-Bezeichnung von der FDA.
- 84% reduction in atrophic lesion growth in treated eyes versus untreated eyes in preliminary Phase 1 data
- EMA orphan medicinal product designation grants 10 years of market exclusivity
- DSMB recommended progression to Phase 2 trials
- Favorable safety and tolerability profile reported in clinical trials
- None.
Insights
The EMA's orphan medicinal product designation for OCU410ST represents a significant milestone for Ocugen's gene therapy program. The preliminary Phase 1/2 data showing an 84% reduction in atrophic lesion growth is particularly impressive for Stargardt disease, which currently has no approved treatments.
The market exclusivity benefit of 10 years in Europe, combined with the existing orphan drug designation in the US, strengthens Ocugen's commercial potential. The completed Phase 1 dosing with positive DSMB recommendation and favorable safety profile indicates strong clinical progress. The addressable market of 100,000 patients in the US and Europe presents a substantial commercial opportunity.
The modifier gene therapy approach using RORA gene delivery shows promise in addressing multiple ABCA4-associated retinopathies. The potential for accelerated MAA could expedite the path to commercialization, though careful monitoring of future trial results remains crucial.
MALVERN, Pa., Nov. 20, 2024 (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 Medicines Agency (EMA) has granted orphan medicinal product designation for OCU410ST for the treatment of ABCA4-associated retinopathies including Stargardt disease, retinitis pigmentosa 19 (RP19), and cone-rod dystrophy 3 (CORD3).
“We are deeply honored to receive orphan medicinal product designation from the EMA for OCU410ST. This recognition brings us one step closer to providing a much-needed option for Stargardt patients who currently have no therapies available,” said Dr. Arun Upadhyay, Chief Scientific Officer and Head of R&D at Ocugen. “We are committed to advancing this treatment with urgency and dedication, with the hope of making a meaningful impact on the lives of those affected by this challenging disease."
The U.S. Food and Drug Administration (FDA) previously granted orphan drug designation to OCU410ST in April 2023. Stargardt disease affects approximately 100,000 people in the U.S. and Europe combined.
Orphan medicinal product designation in Europe offers certain benefits to drug developers while they develop drugs intended for safe and effective treatment, diagnosis, or prevention of rare diseases or conditions that impact fewer than 5 in 10,000 patients in the European Union. Benefits include protocol assistance, reduced regulatory fees, research grants, and 10 years of market exclusivity following regulatory approval.
Dosing in the first phase of the Phase 1/2 OCU410ST GARDian trial for Stargardt disease is complete and the Data and Safety Monitoring Board (DSMB) has recommended moving forward with Phase 2. To date, the safety and tolerability profile of OCU410ST appears to be very favorable.
Preliminary efficacy and safety data from the Phase 1 dose-escalation portion of the Phase 1/2 OCU410ST GARDian clinical trial was recently presented at Ocugen’s Clinical Showcase. Data from evaluable subjects at six months demonstrated a remarkable
“We are encouraged by the preliminary efficacy data showing stabilization or improvement in visual function and retinal structure outcomes in OCU410ST treated eyes,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “These positive clinical and regulatory milestones continue to support the potential for OCU410ST to address inherited retinal diseases with a one-time therapy for life.”
OCU410ST utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene and further represents the impact of Ocugen’s modifier gene therapy approach, which is based on Nuclear Hormone Receptors (NHRs) that regulate diverse physiological functions such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation and cell survival networks.
Ocugen intends to pursue an accelerated marketing authorization application (MAA) for OCU410ST.
About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, 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, strategy, business plans and objectives for Ocugen’s clinical programs, plans and timelines for the preclinical and clinical development of Ocugen’s product candidates, including the therapeutic potential, clinical benefits and safety thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, the ability to initiate new clinical programs; 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; 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 annual and periodic 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 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:
Tiffany Hamilton
Head of Communications
Tiffany.Hamilton@ocugen.com
FAQ
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