Tinlarebant is an investigational, once-daily oral therapy for the treatment of STGD1, a rare, inherited retinal disease caused by mutations in the ABCA4 gene that leads to progressive and irreversible vision loss. STGD1 affects an estimated 9,500 people in Japan alone, and there are currently no approved treatment options for the disease.

Tinlarebant has been designated a SAKIGAKE pharmaceutical product for accelerated review and the NDA in Japan was filed side-by-side with the United States NDA to enable simultaneous early access for patients in both regions. Tinlarebant will be reviewed under the Sakigake Designation System - Japan’s expedited regulatory pathway for innovative medical products. If approved, it could become the first Sakigake pharmaceutical product for ophthalmology disease in Japan.

"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 against our goal to bring tinlarebant to STGD1 patients around the world," said Dr. Tom Lin, Chairman and Chief Executive Officer of Belite Bio. "I'd like to sincerely thank everyone in Japan who participated in or helped facilitate our clinical trials, as well as our entire team who helped make this submission possible. We look forward to working closely with the MHLW as they review our application."

Sakigake designation was established by MHLW to accelerate the drug approval process in Japan for innovative drugs with prominent effectiveness targeting serious diseases, in order to make them available to patients in Japan ahead of the rest of the world, by providing (a) prioritized consultation, (b) pre-application consultation, (c) prioritized review, (d) assignment of a review partner, and (e) extension of re-examination period.

"Although Stargardt disease was first described nearly 120 years ago, no approved treatment has been established to date. 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," said Kaz Tsunaba, President of Belite Bio Japan. "We extend our sincere gratitude to the patients, their families, and the healthcare professionals who have supported the development of this therapy. As tinlarebant could become the first ophthalmology pharmaceutical product to receive regulatory approval under the SAKIGAKE designation, we remain committed to advancing all necessary preparations to ensure that it can be delivered to patients in Japan as early as possible."