Belite Bio’s Tinlarebant Wins FDA Priority Review for Stargardt Disease

The FDA accepted Belite Bio’s New Drug Application for tinlarebant (LBS-008) and granted Priority Review for the treatment of Stargardt Disease Type 1 (STGD1), a rare inherited retinal disorder associated with progressive and irreversible vision loss.

The regulatory filing is based on results from the Phase 3 DRAGON trial (NCT05244304), in which tinlarebant produced a statistically significant and clinically meaningful reduction in the growth rate of atrophic retinal lesions. The company reported a 35.7% reduction versus placebo, measured by definitely decreased autofluorescence (DDAF) using fundus autofluorescence imaging.

If approved, tinlarebant would become the first FDA-approved treatment for STGD1, according to Belite Bio.

Targeting Retinal Bisretinoid Accumulation

STGD1 is caused by mutations in the ABCA4 gene and typically begins early in life. Progressive retinal damage can lead to substantial and irreversible loss of vision. Belite Bio estimates that approximately 53,000 people in the U.S. live with the disease.

Tinlarebant is an oral, once-daily therapy that targets a key component of the visual cycle. The drug reduces serum retinol binding protein 4 (RBP4), the main carrier responsible for transporting retinol from the liver to the eye.

By lowering and maintaining serum RBP4 levels, tinlarebant reduces the amount of retinol reaching the retina. This is intended to limit formation and accumulation of vitamin A-derived bisretinoids, toxic compounds implicated in retinal disease progression.

Phase 3 DRAGON Data Support NDA

The DRAGON study evaluated tinlarebant in patients with STGD1 and provided the pivotal efficacy evidence for the NDA. The primary reported finding was a statistically significant reduction in the rate of DDAF lesion growth compared with placebo.

The company said tinlarebant was generally well tolerated across clinical trials, with adverse effects consistent with its mechanism of action. The announcement did not provide detailed event rates or additional safety data from the Phase 3 study.

The imaging findings are clinically relevant because progressive retinal atrophy represents a central feature of STGD1 and contributes to irreversible visual decline.

Regulatory Path and Next Milestone

Belite Bio said the FDA’s Priority Review reflects the unmet need for an approved treatment in Stargardt disease. Tinlarebant has previously received Breakthrough Therapy, Fast Track and Orphan Drug designations in the U.S., along with Rare Pediatric Disease designation. It also holds Orphan Drug designations in Europe, Japan and Switzerland and Sakigake designation in Japan.

The FDA is scheduled to complete its review by February 12, 2027, the NDA’s PDUFA date.

Beyond STGD1, tinlarebant is being evaluated as a potential treatment for geographic atrophy associated with advanced dry age-related macular degeneration, where bisretinoid accumulation also contributes to retinal disease. The upcoming FDA decision will determine whether tinlarebant becomes the first approved pharmacologic treatment specifically for Stargardt Disease Type 1.

Reference

Belite Bio Announces U.S. Food and Drug Administration Acceptance and Priority Review of New Drug Application for Tinlarebant for the Treatment of Stargardt Disease Type 1 | Belite Bio, Inc.