The US Food and Drug Administration (FDA) has granted priority review designation for the new drug application for tinlarebant (LBS-008; Belite Bio) for the treatment of Stargardt Disease Type 1 (STGD1). The FDA has set a Prescription Drug User Fee Act date of February 12, 2027.

According to the company, the filing was based on the results from the phase 3 DRAGON trial, which evaluated tinlarebant for the treatment of STGD1. In the study, tinlarebant demonstrated a statistically significant and clinically meaningful 35.7% reduction in the growth rate of atrophic retinal lesions, measured as definitely decreased autofluorescence by fundus autofluorescence imaging, compared with placebo. Tinlarebant has been generally well tolerated in clinical trials with side effects consistent with its mechanism of action. If approved, tinlarebant would be the first FDA-approved treatment option for STGD1, the company said in the press release. 

Tinlarebant is a novel oral therapy that is intended to reduce the accumulation of vitamin A-based toxins (known as bisretinoids) that cause retinal disease in Stargardt Disease and also contribute to disease progression in geographic atrophy, or advanced dry age-related macular degeneration. Bisretinoids are by-products of the visual cycle, which is dependent on the supply of vitamin A to the eye. Tinlarebant works by reducing and maintaining levels of serum retinol binding protein 4, the sole carrier protein for retinol transport from the liver to the eye. By modulating the amount of retinol entering the eye, tinlarebant reduces the formation of bisretinoids.

Tinlarebant has been granted Breakthrough Therapy Designation, Fast Track Designation, and Rare Pediatric Disease Designation in the US, Orphan Drug Designation in the US, Europe, Japan, and Switzerland, and Sakigake Designation in Japan for the treatment of Stargardt Disease. RP