Belite Bio files Japan NDA for tinlarebant in Stargardt disease type 1

Belite Bio, Inc announced on September 8, 2026 that it had submitted a New Drug Application to Japan's Ministry of Health, Labour, and Welfare for tinlarebant as a treatment for Stargardt disease type 1.¹

The company noted that the FDA had already accepted a New Drug Application for tinlarebant under priority review, with a Prescription Drug User Fee Act target action date of February 12, 2027.¹ Belite Bio said that if approved, tinlarebant would be the first treatment available for Stargardt Disease Type 1, and could also become the first Sakigake-designated ophthalmology product in Japan.¹

Tinlarebant is described as an investigational, once-daily oral therapy meant for STGD1, a rare inherited retinal condition caused by mutations in the ABCA4 gene that results in progressive, irreversible vision loss.¹ The company said the condition affects roughly 9,500 people in Japan, where no approved treatments currently exist.¹

Belite Bio said the drug carries Sakigake designation for accelerated review, and that the Japanese filing was submitted in parallel with the U.S. application to allow for simultaneous early patient access in both markets.¹ The Sakigake pathway, according to the company, was created by MHLW to speed approval of innovative drugs for serious diseases, offering benefits such as prioritized consultation, pre-application consultation, prioritized review, an assigned review partner, and an extended re-examination period.¹

On the clinical side, Belite Bio said its Phase 3 DRAGON trial in adolescent and adult STGD1 patients achieved its primary endpoint, while tinlarebant continues to be studied in the Phase 2/3 DRAGON II trial for STGD1 and the Phase 3 PHOENIX trial for geographic atrophy.¹ Tinlarebant has also received Breakthrough Therapy, Fast Track, and Rare Pediatric Disease designations in the U.S., plus Orphan Drug status in the U.S., Europe, Japan, and Switzerland.¹