Belite Bio Submits NDA for Tinlarebant in Japan

Belite Bio, a clinical-stage drug development company focused on advancing novel therapeutics for degenerative retinal diseases, has submitted a New Drug Application (NDA) to the Ministry of Health, Labour, and Welfare (MHLW) in Japan for its investigational, once-daily oral therapy, tinlarebant. This submission marks a significant milestone in the company's efforts to bring a potential breakthrough treatment for Stargardt disease type 1 (STGD1) to patients in Japan and the United States.

Tinlarebant is designed to reduce the accumulation of vitamin A-based toxins, known as bisretinoids, which contribute to the progressive vision loss associated with STGD1. The disease, caused by mutations in the ABCA4 gene, affects approximately 9,500 people in Japan and currently has no approved treatment options.

The submission of the NDA to the MHLW in Japan is part of a broader strategy to secure early access for patients in both regions. Tinlarebant has been designated a Sakigake pharmaceutical product, an expedited regulatory pathway aimed at making innovative drugs with significant effectiveness for serious diseases available to patients ahead of the rest of the world. This designation includes prioritized consultation, pre-application consultation, prioritized review, assignment of a review partner, and extension of the re-examination period.

Dr. Tom Lin, Chairman and CEO of Belite Bio, expressed the significance of this submission: "The NDA submission in Japan under Sakigake designation reflects the significant unmet need for people living with STGD1 and is backed by our robust data showing tinlarebant’s ability to slow the growth rate of retinal lesions compared to placebo. This is another important milestone as we continue to make rapid progress toward bringing tinlarebant to STGD1 patients around the world."

The company has already achieved notable progress with tinlarebant. In the U.S., the New Drug Application (NDA) for tinlarebant for the treatment of STGD1 was accepted by the U.S. Food and Drug Administration (FDA) with Priority Review. The Prescription Drug User Fee Act (PDUFA) target action date for this application is February 12, 2027. If approved, tinlarebant would be the first-ever approved treatment for STGD1 and could become the first Sakigake pharmaceutical product for ophthalmology disease in Japan.

Kaz Tsunaba, President of Belite Bio Japan, echoed Dr. Lin's sentiments, emphasizing the company's commitment to bringing tinlarebant to patients in Japan as early as possible. "We view our NDA in Japan as an important step toward delivering the first therapeutic option in the country for people living with Stargardt disease," he said.

The clinical development of tinlarebant includes a Phase 3 trial (DRAGON) in adolescent and adult subjects with STGD1, which met its primary endpoint. The drug is also being evaluated in a Phase 2/3 trial (DRAGON II) for adolescents and adults with STGD1 and a Phase 3 trial (PHOENIX) for subjects with geographic atrophy (GA), a form of advanced dry age-related macular degeneration (AMD).

Belite Bio's lead candidate, tinlarebant, has received various designations, including Breakthrough Therapy Designation, Fast Track Designation, and Rare Pediatric Disease Designation in the U.S., as well as Orphan Drug Designation in the U.S., Europe, Japan, and Switzerland. The company remains committed to advancing all necessary preparations to ensure that tinlarebant can be delivered to patients as soon as possible.