Belite Bio has completed a rolling new drug application (NDA) submission to the US Food and Drug Administration (FDA) for tinlarebant, an investigational oral therapy for Stargardt disease type 1, the company announced.

The application was submitted under the FDA’s Breakthrough Therapy program and concludes a rolling review process that began in April. The agency will now conduct a 60-day filing review to determine whether the application is sufficiently complete for formal review. If accepted, the FDA will assign a Prescription Drug User Fee Act (PDUFA) target action date.

Tinlarebant is being developed for Stargardt disease type 1, an inherited retinal disorder caused by mutations in the ABCA4 gene. The disease is characterized by progressive vision loss and currently has no approved treatments in the United States.

The NDA is supported by data from the phase 3 DRAGON trial, which met its primary endpoint. According to Belite Bio, patients treated with tinlarebant experienced a significant reduction in the growth rate of retinal lesions compared with placebo. The therapy is administered orally once daily.

Tinlarebant is designed to reduce the accumulation of bisretinoids, vitamin A–derived byproducts that contribute to retinal degeneration in Stargardt disease. The drug lowers levels of retinol binding protein 4, thereby reducing the amount of vitamin A delivered to the eye and limiting bisretinoid formation.

In addition to Breakthrough Therapy Designation, tinlarebant has received Fast Track, Rare Pediatric Disease, and Orphan Drug designations in the United States. The drug is also being evaluated in the phase 2/3 DRAGON II study in Stargardt disease and the phase 3 PHOENIX trial in geographic atrophy associated with age-related macular degeneration.