The FDA has accepted Belite Bio’s application for tinlarebant and granted Priority Review for the potential treatment of Stargardt disease type 1.

FDA sets February 2027 decision date

The FDA has accepted Belite Bio’s New Drug Application (NDA) for tinlarebant for the treatment of Stargardt disease type 1 (STGD1) and granted the medicine Priority Review.

The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of February 12, 2027.

If approved, tinlarebant would become the first FDA-approved treatment for STGD1, a rare inherited retinal disease caused by mutations in the ABCA4 gene.

STGD1 typically begins early in life and causes progressive, irreversible vision loss. An estimated 53,000 people in the US are affected by the disease.

The NDA is supported by results from the Phase 3 DRAGON trial, which evaluated tinlarebant in adolescents and adults with STGD1.

Phase 3 trial showed reduced retinal lesion growth

In the DRAGON trial, tinlarebant reduced the growth rate of atrophic retinal lesions by 35.7% compared with placebo.

The result was measured using definitely decreased autofluorescence (DDAF) through fundus autofluorescence imaging and met the study’s primary endpoint.

Tinlarebant is an oral, once-daily treatment designed to reduce the accumulation of vitamin A-derived toxins called bisretinoids in the retina.

Bisretinoids are formed as by-products of the visual cycle and accumulate in the eye in conditions including Stargardt disease.

Belite Bio said tinlarebant works by reducing and maintaining levels of retinol-binding protein 4 (RBP4), the carrier protein responsible for transporting vitamin A from the liver to the eye.

By reducing the amount of retinol entering the eye, the treatment is intended to reduce the formation of bisretinoids.

Belite Bio chief executive Tom Lin said: “The acceptance of our NDA with Priority Review designation underscores the immediate need among the Stargardt disease community for an approved treatment option.”

Lin added that the company would work with the FDA through the review process.

Potential first approved treatment for STGD1

There are currently no approved disease-modifying treatments for Stargardt disease type 1.

Existing management can include visual aids and other measures to help people adapt to progressive vision loss, but these approaches do not target the underlying disease mechanism.

The potential approval of tinlarebant would therefore represent a new treatment option for people living with STGD1.

Belite Bio chief medical officer Hendrik Scholl said: “As a practicing physician, I have treated people living with Stargardt disease for more than 20 years and have seen firsthand the challenges that it brings.”

Scholl added that current options as vision deteriorates are limited to visual aids, highlighting the potential importance of a treatment targeting the underlying disease process.

Tinlarebant has previously received several regulatory designations in the US, Europe and Japan, including Breakthrough Therapy, Fast Track, Rare Pediatric Disease and Orphan Drug designations.

The FDA’s Priority Review designation means the application will receive a shorter review target than a standard application. The agency will make its decision by the February 12, 2027 PDUFA date unless the review timeline changes.

Belite Bio is also evaluating tinlarebant in DRAGON II, a Phase 2/3 trial in adolescent and adult patients with STGD1, and in the Phase 3 PHOENIX trial in people with geographic atrophy associated with advanced dry age-related macular degeneration.