Ocugen, Inc. Announces FDA Approval of Expanded Access Program for Patients with Retinitis Pigmentosa
Ocugen, Inc. (NASDAQ: OCGN) has received FDA approval to begin an expanded access program (EAP) for OCU400, its modifier gene therapy candidate for retinitis pigmentosa (RP). The EAP will allow adult patients aged 18 and older with RP to access OCU400 outside of clinical trials. This development follows positive Phase 1/2 study data and an ongoing Phase 3 liMeliGhT clinical trial.
OCU400 has received orphan drug and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA. The European Medicines Agency (EMA) has also accepted the U.S.-based trial for submission of a Marketing Authorization Application (MAA). Ocugen aims for targeted BLA and MAA approval in 2026.
Ocugen, Inc. (NASDAQ: OCGN) ha ricevuto l'approvazione della FDA per avviare un programma di accesso espanso (EAP) per OCU400, il suo candidato per la terapia genica modificante per la retinite pigmentosa (RP). L'EAP consentirà ai pazienti adulti di età pari o superiore a 18 anni affetti da RP di accedere a OCU400 al di fuori degli studi clinici. Questo sviluppo segue i dati positivi dello studio di Fase 1/2 e un trial clinico di Fase 3 chiamato liMeliGhT in corso.
OCU400 ha ricevuto designazioni di farmaco orfano e di Terapia Avanzata di Medicina Rigenerativa (RMAT) da parte della FDA. Anche l'Agenzia Europea dei Medicinali (EMA) ha accettato il trial basato negli Stati Uniti per la presentazione di una Domanda di Autorizzazione al Mercato (MAA). Ocugen punta a ottenere l'approvazione mirata della BLA e della MAA entro il 2026.
Ocugen, Inc. (NASDAQ: OCGN) ha recibido la aprobación de la FDA para iniciar un programa de acceso expandido (EAP) para OCU400, su candidato a terapia génica modificadora para la retinitis pigmentosa (RP). El EAP permitirá a los pacientes adultos de 18 años o más con RP acceder a OCU400 fuera de ensayos clínicos. Este desarrollo sigue a datos positivos del estudio de Fase 1/2 y a un ensayo clínico de Fase 3 en curso llamado liMeliGhT.
OCU400 ha recibido designaciones de medicamento huérfano y de Terapia Avanzada en Medicina Regenerativa (RMAT) por parte de la FDA. La Agencia Europea de Medicamentos (EMA) también ha aceptado el ensayo basado en EE. UU. para la presentación de una Solicitud de Autorización de Comercialización (MAA). Ocugen tiene como objetivo obtener la aprobación puntual de la BLA y la MAA en 2026.
Ocugen, Inc. (NASDAQ: OCGN)는 확장 접근 프로그램(EAP)을 시작하기 위해 FDA의 승인을 받았습니다. 이 프로그램은 망막색소변성증(RP) 치료를 위한 유전자 치료 후보인 OCU400에 대해 다룹니다. EAP는 18세 이상의 성인 RP 환자들이 임상 시험 외부에서 OCU400에 접근할 수 있도록 허용합니다. 이 발전은 1/2상 연구의 긍정적인 데이터와 진행 중인 3상 liMeliGhT 임상 시험을 따릅니다.
OCU400은 FDA로부터 고아 약물 및 재생 의학 고급 치료(RMAT) 지정도 받았습니다. 유럽 의약청(EMA) 역시 미국 기반 임상시험의 마케팅 승인 신청(MAA)을 승인했습니다. Ocugen은 2026년 내에 BLA 및 MAA 승인을 목표로 하고 있습니다.
Ocugen, Inc. (NASDAQ: OCGN) a reçu l'approbation de la FDA pour commencer un programme d'accès élargi (EAP) pour OCU400, son candidat à la thérapie génique modificateur pour la rétinite pigmentaire (RP). L'EAP permettra aux patients adultes de 18 ans et plus atteints de RP d'accéder à OCU400 en dehors des essais cliniques. Ce développement fait suite à des données positives d'étude de phase 1/2 et à un essai clinique de phase 3 en cours appelé liMeliGhT.
OCU400 a obtenu les désignations de médicament orphelin et de thérapie avancée en médecine régénérative (RMAT) de la FDA. L'Agence européenne des médicaments (EMA) a également accepté l'essai basé aux États-Unis pour la soumission d'une demande d'autorisation de mise sur le marché (MAA). Ocugen vise une approbation ciblée de la BLA et de la MAA en 2026.
Ocugen, Inc. (NASDAQ: OCGN) hat die Genehmigung der FDA erhalten, ein erweitertes Zugangsprogramm (EAP) für OCU400, seinen modifizierenden Gentherapie-Kandidaten für die Retinitis pigmentosa (RP), zu starten. Das EAP ermöglicht es erwachsenen Patienten ab 18 Jahren mit RP, außerhalb von klinischen Studien auf OCU400 zuzugreifen. Diese Entwicklung folgt positiven Daten aus der Phase 1/2-Studie und einer laufenden Phase-3-Studie namens liMeliGhT.
OCU400 hat von der FDA die Auszeichnung als Orphan Drug und als Regenerative Medicine Advanced Therapy (RMAT) erhalten. Die Europäische Arzneimittel-Agentur (EMA) hat auch die Einreichung eines Antrags auf Marktzulassung (MAA) für die in den USA durchgeführte Studie akzeptiert. Ocugen strebt die genehmigte BLA und MAA bis 2026 an.
- FDA approval for expanded access program (EAP) for OCU400 in retinitis pigmentosa patients
- Positive Phase 1/2 study data and ongoing Phase 3 clinical trial
- OCU400 received orphan drug and RMAT designations from FDA
- EMA accepted U.S.-based trial for MAA submission
- Targeted BLA and MAA approval in 2026
- None.
The FDA's approval of Ocugen's Expanded Access Program (EAP) for OCU400 is a significant milestone in the treatment of Retinitis Pigmentosa (RP). This decision allows patients with unmet medical needs to access the experimental gene therapy before official FDA approval, potentially benefiting up to
The EAP approval, coupled with the ongoing Phase 3 liMeliGhT clinical trial, suggests growing confidence in OCU400's safety and efficacy. The therapy's modifier gene approach could potentially address multiple RP gene mutations, offering a broader treatment scope than current gene-specific therapies. This could be a game-changer for RP patients, especially those with rare mutations lacking targeted treatments.
However, investors should note that EAP approval doesn't guarantee final FDA approval. The 2026 target for BLA and MAA approval indicates a long road ahead, with potential risks and uncertainties in the clinical development process.
Ocugen's EAP approval for OCU400 is a positive development for the company and its shareholders. This milestone could potentially accelerate revenue generation before full FDA approval, albeit on a scale. The EAP also provides an opportunity to gather additional real-world data, which could support the ongoing Phase 3 trial and future regulatory submissions.
The company's multi-pronged approach, including orphan drug and RMAT designations from the FDA and EMA's acceptance of the U.S.-based trial for MAA submission, demonstrates a strategic regulatory pathway. This could potentially expedite market entry in both the U.S. and Europe, expanding Ocugen's market reach.
However, investors should consider the financial implications of running an EAP alongside a Phase 3 trial. While this could provide valuable data, it may also increase operational costs. The
MALVERN, Pa., Aug. 05, 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 and vaccines, today announced that it has received notification from FDA to begin its expanded access program (EAP) for the treatment of adult patients, aged 18 and older, with retinitis pigmentosa (RP) with OCU400—a modifier gene therapy product candidate.
“Each clinical milestone achieved by OCU400 brings us closer to providing a potential one-time treatment for life to patients living with RP,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “With positive Phase 1/2 study data and an ongoing Phase 3 liMeliGhT (pronounced “limelight”) clinical trial, we now plan to work with clinicians, patients, and the RP community to provide access to OCU400 for eligible patients through our EAP. The EAP strengthens our commitment to serving RP patients—300,000 in the U.S. and Europe and 1.6 million globally.”
EAP allows patients who have unmet medical needs with serious or life-threatening conditions to access treatments outside of a clinical trial that are not yet approved by the FDA.
The OCU400 EAP is available for patients with early, intermediate to advanced RP with at least minimal retinal preservation who may benefit from the mechanism of action of OCU400 prior to approval of the Biologics License Application (BLA). Ocugen is actively dosing patients in the Phase 3 liMeliGhT clinical trial.
“RP patients with mutations in multiple genes currently have no therapeutic options. As a retinal surgeon, I am encouraged by the therapeutic potential of OCU400 to provide long-term benefit,” said Lejla Vajzovic, MD, FASRS, Director, Duke Surgical Vitreoretinal Fellowship Program, Associate Professor of Ophthalmology with Tenure, Adult and Pediatric Vitreoretinal Surgery and Disease, Duke University Eye Center, and Retina Scientific Advisory Board Chair of Ocugen. “The OCU400 EAP gives RP patients access to this novel modifier gene therapy outside of the ongoing Phase 3 study.”
“We are pleased to make OCU400 available to patients beyond our Phase 3 liMeliGhT clinical trial through this EAP,” said Dr. Huma Qamar, Ocugen’s Chief Medical Officer. "We are excited to expand our enrollment to include patients representing a diverse array of RP gene mutations. This program reflects our ongoing commitment to develop a safe and effective therapy for RP patients who may not have other treatment options.”
Ocugen previously announced that OCU400 has received orphan drug and Regenerative Medicine Advanced Therapy (RMAT) designations from FDA and that the European Medicines Agency (EMA) accepted the U.S.-based trial for submission of a Marketing Authorization Application (MAA). With the dosing of patients in the Phase 3 clinical trial program underway, OCU400 remains on track for targeted BLA and MAA approval in 2026.
About OCU400 EAP
The OCU400 EAP is a U.S.-only protocol for (1) eligible adult RP patients, 18 years and older, with early, intermediate to advanced disease with at least minimal retinal preservation, (2) patients who participated in the OCU400 Phase 1/2 study and who qualify for dosing in the contralateral eye, (3) patients who failed to meet inclusion criteria in the Phase 1/2 trial and ongoing Phase 3 liMeliGhT clinical trial who could benefit from OCU400, and (4) RP patients who can benefit from the mechanism of action of OCU400 prior to BLA approval.
Additional information on the OCU400 EAP will be available on www.clinicaltrials.gov.
About OCU400 Phase 3 (liMeliGhT) for RP
The Phase 3 liMeliGhT clinical trial, with a duration of one year, will have a sample size of 150 participants. One arm will include 75 participants with RHO gene mutations, and the other arm will include 75 participants who have mutations in other genes. Within each arm, participants will be randomized 2:1 to the treatment group (2.5 x1010 vector genomes/eye of OCU400) and untreated control group, respectively. Patients eight years of age and older with early to late-stage RP are being recruited to participate in the liMeliGhT study.
About OCU400
OCU400 is the Company’s modifier gene therapy product based on a nuclear hormone receptor (NHR) gene called NR2E3. This gene regulates diverse physiological functions within the retina, such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation, and cell survival. Retinal cells in RP patients have a dysfunctional gene network, and OCU400 resets this network to reestablish a healthy cellular homeostasis—which has the potential to improve vision in patients with RP.
About Modifier Gene Therapy
Modifier gene therapy is designed to fulfill unmet medical needs related to retinal diseases, including IRDs, such as RP, Leber congenital amaurosis (LCA) and Stargardt disease, as well as multifactorial diseases like dry age-related macular degeneration (dAMD). Our modifier gene therapy platform is based on the use of NHRs, master gene regulators, which have the potential to restore homeostasis — the basic biological processes in the retina. Unlike single-gene replacement therapies, which only target one genetic mutation, we believe that our modifier gene therapy platform, through its use of NHRs, represents a novel approach that has the potential to address multiple retinal diseases caused by mutations in multiple genes with one product, and to address complex diseases that are potentially caused by imbalances in multiple gene networks. Currently, Ocugen has three modifier gene therapy programs in the clinic: OCU400, OCU410, and OCU410ST. In addition to the OCU400 Phase 3 liMeliGhT clinical trial, the OCU410 Phase 1/2 ArMaDa clinical trial for geographic atrophy (GA) secondary to dAMD and the OCU410ST Phase 1/2 GARDian clinical trial for Stargardt disease are currently underway. GA affects approximately two to three million people in the U.S. and EU combined and Stargardt disease affects nearly 100,000 people in the U.S. and EU combined.
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 patients’ 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; 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 “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 Corporate Communications
Tiffany.Hamilton@ocugen.com
FAQ
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