GlobeNewswire·Sep 8, 2026, 6:00 AM ET

Belite Bio Announces Submission of New Drug Application to the Ministry of Health, Labour, and Welfare in Japan under the Sakigake Designation System for Tinlarebant for the Treatment of Stargardt Disease Type 1

Belite Bio submitted a New Drug Application (NDA) for tinlarebant to Japan's Ministry of Health, Labour, and Welfare for Stargardt disease type 1.
The company also filed a parallel NDA in the U.S., which the FDA accepted with Priority Review and a Prescription Drug User Fee Act target action date of February 12, 2027.
If approved, tinlarebant would be the first-ever treatment for Stargardt disease type 1 in Japan.
The drug has been designated as a SAKIGAKE pharmaceutical product in Japan for accelerated review.
Tinlarebant is an investigational, once-daily oral therapy intended to reduce the accumulation of vitamin A-based toxins that cause retinal disease.
The NDA submission was supported by Phase 3 trial (DRAGON) data that met its primary endpoint showing a slowed growth rate of retinal lesions.
Stargardt disease type 1 affects approximately 9,500 people in Japan, with no currently approved treatment options available.
The drug holds multiple designations including Breakthrough Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug Designation in the U.S., Europe, Japan, and Switzerland.
Belite Bio is also evaluating tinlarebant in a Phase 2/3 trial (DRAGON II) for Stargardt disease type 1 and a Phase 3 trial (PHOENIX) for geographic atrophy.