Belite Bio to Seek FDA Approval for its Stargardt Disease Drug

Belite Bio, a biopharmaceutical company developing drugs for retinal degenerative diseases, plans to seek approval from the US Food & Drug Administration (FDA) for Tinlarebant, its oral drug designed to slow disease progression and vision loss for people with Stargardt disease. The company plans to file a new drug application (NDA) for the drug with the FDA in the first half of 2026. Belite is also seeking regulatory approval for Tinlarebant in the UK and China.

In its global Phase 3 Dragon clinical trial, Tinlarebant reduced the growth rate of atrophic lesions (areas of central retinal cell loss) by 36 percent for patients receiving the drug compared to those getting a placebo. Visual acuity for patients in Dragon was stable across the trial, regardless of treatment group, which was expected given the natural history of Stargardt disease for the population enrolled. The 104 patients (ages 12-20) in the trial were randomized two to one to receive Tinlarebant or a placebo. Results were reported after 24 months of treatment. 

Tinlarebant is designed to inhibit retinol binding protein 4 (RBP4) to reduce the uptake of vitamin A into the retina, thereby decreasing the production and accumulation of toxic byproducts from vitamin A processing, which are the hallmark of Stargardt disease. The condition is usually caused by mutations in the gene ABCA4. Stargardt disease affects approximately 30,000 people in the US. While the severity and rate of progression of the condition can vary, it usually causes significant central vision loss.

ProgStar, the largest-ever natural history study of patients with Stargardt disease, was used to inform the Dragon clinical trial design. Funded by the Foundation, ProgStar was launched in 2013 and prospectively followed 259 patients for two years. The study’s retrospective arm enrolled 251 patients.

Belite is currently enrolling Stargardt disease patients (ages 12-20) in its Phase 2/3 Dragon II clinical trial for Tinlarebant, taking place at sites in the US, UK, and Japan.

The company has also fully enrolled its global Phase 3 Phoenix clinical trial for Tinlarebant for patients with geographic atrophy, the advanced form of dry age-related macular degeneration.