FDA Grants Priority Review to Tinlarebant for Stargardt Disease Type 1

The U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review for Belite Bio's New Drug Application (NDA) for tinlarebant, an investigational oral therapy for Stargardt disease type 1 (STGD1). If approved, tinlarebant would be the first FDA-authorized treatment for this rare inherited retinal dystrophy, which currently lacks pharmacologic options. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of February 12, 2027. The NDA submission is based on data from the Phase 3 DRAGON trial, which showed a statistically significant 35.7% reduction in the growth rate of atrophic retinal lesions compared to placebo.