Belite Bio Says FDA Grants Priority Review to Stargardt Disease Drug

By Anvee Bhutani

Belite Bio said Tuesday that the U.S. Food and Drug Administration accepted its application for tinlarebant, an experimental treatment for Stargardt disease type 1, and granted the drug Priority Review.

The FDA set a target action date of Feb. 12, 2027, the San Diego-based biotechnology company said.

The application was supported by results from a Phase 3 trial, in which tinlarebant reduced the growth rate of atrophic retinal lesions by 35.7% compared with placebo, Belite said. The company said the drug was generally well tolerated in clinical trials.

If approved, tinlarebant would be the first FDA-approved treatment for Stargardt disease type 1, a rare inherited retinal disease caused by mutations in the ABCA4 gene that can lead to progressive and irreversible vision loss.

Stargardt disease affects an estimated 53,000 people in the U.S., according to Belite.

Tinlarebant is an oral, once-daily drug designed to reduce the formation of bisretinoids, vitamin A-derived toxins that accumulate in the retina and contribute to retinal degeneration, the company said.

The drug has previously received Breakthrough Therapy, Fast Track and Rare Pediatric Disease designations from the FDA, as well as Orphan Drug designation in the U.S., Europe, Japan and Switzerland, Belite said.

Write to Anvee Bhutani at anvee.bhutani@wsj.com

(END) Dow Jones Newswires

August 11, 2026 17:34 ET (21:34 GMT)