Belite Bio, Inc. (NASDAQ: BLTE) submitted a New Drug Application to Japan's Ministry of Health, Labour, and Welfare for tinlarebant, an oral therapy for Stargardt disease type 1. The Japanese application was filed simultaneously with the company's U.S. submission.
Stargardt disease type 1 is a rare inherited retinal disease caused by mutations in the ABCA4 gene, leading to progressive vision loss. The condition affects approximately 9,500 people in Japan, and there are currently no approved treatments.
Tinlarebant received Sakigake designation in Japan, an expedited regulatory pathway for medical products targeting serious diseases. In the United States, the Food and Drug Administration accepted the company's New Drug Application with Priority Review and set a target action date of February 12, 2027, under the Prescription Drug User Fee Act. The drug also holds Breakthrough Therapy Designation, Fast Track Designation and Rare Pediatric Disease Designation in the U.S., as well as orphan drug designations in the United States, Europe, Japan and Switzerland.
The therapy works by reducing levels of serum retinol binding protein 4, a protein that transports vitamin A from the liver to the eye. By lowering that protein, tinlarebant is intended to reduce the formation of bisretinoids, toxic by-products that accumulate in the retina.
Belite Bio completed a Phase 3 trial called DRAGON in adolescent and adult subjects with Stargardt disease type 1, which met its primary endpoint. The company is also conducting additional trials, including DRAGON II and a Phase 3 trial called PHOENIX for geographic atrophy.