Ocugen announced the US Food and Drug Administration (FDA) has granted rare paediatric disease designation for OCU410ST, the company’s gene therapy for ABCA4-associated retinopathies including Stargardt disease, retinitis pigmentosa 19 and cone-rod dystrophy 3.  

In 2019, OCU410ST received orphan drug designations for the treatment of ABCA4-associated retinopathies from the FDA and European Medicines Agency. 

OCU410ST uses an adeno-associated virus delivery platform for the retinal delivery of the RAR-Related Orphan Receptor A gene, which regulates pathophysiological pathways linked to Stargardt disease, such as lipofuscin formation, oxidative stress, complement formation, inflammation and cell survival networks. 

This designation reaffirms the urgency of providing a therapeutic option to Stargardt patients who have no FDA-approved treatment available, said Dr Shankar Musunuri, Ocugen chair, CEO and co-founder. “This inherited retinal disease presents itself most often in childhood, making Stargardt disease a diagnosis that not only affects the patient but impacts the entire family.” 

Ocugen said it may be awarded a priority review voucher (PRV) – designed to incentivise drug development for serious rare paediatric diseases – if the PRV programme is reauthorised by the US Congress. A PRV can be redeemed to receive priority review for a different product or sold to another sponsor, typically for around US$100 million. 

However, Congress failed to reauthorise the programme when it expired in December 2024. Combined with US president Donald Trump’s 10,000 layoffs across health departments, including the FDA, the programme’s lapse could contribute to a slowdown in investment in rare paediatric drug development, said David Ridley, a health economist at Duke University’s Fuqua School of Business, North Carolina.  

Ocugen said it plans to initiate OCU410ST’s phase 2/3 pivotal confirmatory trial imminently and apply for a distribution licence in 2027.