FDA Grants Priority Review to Belite Bio for Stargardt Treatment Tinlarebant

FDA Grants Priority Review to Belite Bio for Stargardt Treatment Tinlarebant

According to reports from Investing.com Nigeria, the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review designation to Belite Bio’s New Drug Application for tinlarebant, an investigational treatment for Stargardt Disease Type 1. For the inherited retinal disease (IRD) community, this regulatory milestone represents a significant step forward as patients and families continue to await the first approved treatment options for this condition.

The submission is supported by data from the Phase 3 DRAGON trial, which evaluated tinlarebant in adolescent and adult subjects. According to the source material, the trial demonstrated a 35.7% reduction in the growth rate of atrophic retinal lesions compared to placebo, as measured by definitely decreased autofluorescence using fundus autofluorescence imaging. Tinlarebant is designed as an oral therapy that aims to reduce the accumulation of vitamin A-based toxins known as bisretinoids, which drive retinal disease in Stargardt patients.

Stargardt Disease Type 1 is a rare inherited retinal condition caused by mutations in the ABCA4 gene, leading to progressive and irreversible vision loss. Belite Bio estimates that the disease impacts approximately 53,000 individuals in the United States. Currently, there are no FDA-approved treatments available for Stargardt disease, leaving those affected with limited management options.

With Priority Review granted, the FDA has set a target action date of February 12, 2027, under the Prescription Drug User Fee Act. As the review process moves forward, the IRD community will be closely watching for the final decision, which will determine whether tinlarebant becomes the first-ever approved treatment for Stargardt Disease Type 1.