Belite Bio reported positive top‑line results from its Phase III DRAGON study: oral tinlarebant slowed the growth rate of retinal lesions in adolescents with Stargardt disease type 1, meeting the trial’s primary endpoint. The trial produced a statistically significant reduction in lesion progression versus placebo. Tinlarebant acts by lowering serum retinol‑binding protein 4 and the company said the data support filings in multiple jurisdictions, including plans to pursue U.S. approval. The result would mark the first pivotal success in a disease with no approved therapies. If global submissions follow, the outcome could change the care pathway for STGD1 patients and create a first‑in‑class oral option for a predominantly pediatric population, with implications for rare‑disease regulatory strategy and access planning.