Ocugen, Inc. has announced that the European Medicines Agency (EMA) has granted orphan medicinal product designation to OCU410ST for the treatment of ABCA4-associated retinopathies, including Stargardt disease, retinitis pigmentosa 19 (RP19), and cone-rod dystrophy 3 (CORD3). This regulatory milestone supports the development of a potential therapy for a group of inherited retinal diseases with limited treatment options.
EMA Orphan Drug Designation
The orphan medicinal product designation by the EMA provides Ocugen with several benefits, including protocol assistance, reduced regulatory fees, access to research grants, and 10 years of market exclusivity upon regulatory approval in the European Union. This designation is reserved for drugs intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or conditions affecting fewer than 5 in 10,000 patients in the EU.
Clinical Trial Progress
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. The safety and tolerability profile of OCU410ST has been favorable to date.
Promising Early Efficacy Data
Preliminary efficacy and safety data from the Phase 1 dose-escalation portion of the Phase 1/2 OCU410ST GARDian clinical trial were recently presented at Ocugen’s Clinical Showcase. Data from evaluable subjects at six months demonstrated an 84% reduction in atrophic lesion growth in treated eyes versus untreated fellow eyes.
Dr. Huma Qamar, Chief Medical Officer at Ocugen, stated, "We are encouraged by the preliminary efficacy data showing stabilization or improvement in visual function and retinal structure outcomes in OCU410ST treated eyes. 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."
Mechanism of Action
OCU410ST utilizes an adeno-associated virus (AAV) delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene. This modifier gene therapy approach 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.
Future Plans
Ocugen intends to pursue an accelerated marketing authorization application (MAA) for OCU410ST, aiming to expedite the availability of this potential therapy to patients in need. 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.
