Belite Bio Inc (NASDAQ: BLTE) announced that the Swiss Agency for Therapeutic Products granted orphan drug status to tinlarebant for treating Stargardt disease, a rare inherited retinal disorder.

The designation provides regulatory and financial incentives including 15 years of document protection compared to the standard 10 years, accelerated review timelines, fee reductions, and potential access to an early access pathway for patients.

Tinlarebant previously met the primary endpoint in the Phase 3 DRAGON trial, demonstrating a 35.7% reduction in retinal lesion growth rate compared to placebo. The trial enrolled 104 subjects across 11 jurisdictions worldwide in a randomized, double-masked, placebo-controlled study of adolescent patients with Stargardt disease.

The drug has received multiple regulatory designations including Breakthrough Therapy, Fast Track and Rare Pediatric Disease Designations in the U.S., along with Orphan Drug Designation in the U.S., Europe, Japan, and now Switzerland. It also received Pioneer Drug Designation in Japan for Stargardt disease treatment.

Belite Bio initiated a rolling submission of a New Drug Application with the U.S. Food and Drug Administration and expects to complete the submission in the second quarter of 2026. The company stated this represents the first therapeutic candidate to demonstrate clinical efficacy in Stargardt disease.

Tinlarebant is an oral therapy designed to reduce vitamin A-based toxin accumulation that causes retinal disease by modulating retinol binding protein 4, which transports vitamin A from the liver to the eye. The drug is also being evaluated in trials for geographic atrophy.