Belite Bio Announces China NMPA Grants Priority Review For New Drug Application Of Tinlarebant In Stargardt Disease Based On Interim Analysis Results

Belite Bio, Inc., a clinical-stage biopharmaceutical company developing novel therapies for degenerative retinal diseases with significant unmet medical needs, announced that China’s National Medical Products Administration (NMPA) has agreed to accept the New Drug Application (NDA) for Tinlarebant with priority review for the treatment of Stargardt disease. This acceptance is based on interim results from the Phase 3 DRAGON trial.

Dr. Tom Lin, Chairman and CEO of Belite Bio, commented, “Having the China NMPA agree to review the NDA based on interim Phase 3 data is a remarkable milestone for Belite Bio and the Stargardt community. This achievement highlights the strength of our program and the urgent need for therapies for this devastating disease, which currently has no approved treatment options. It positions Belite Bio to advance Tinlarebant through the final stages of development and, if successful, deliver the first treatment to people living with Stargardt disease.”

The NMPA’s decision is based on interim analysis showing statistically significant results for the primary endpoint in the Phase 3 DRAGON trial. Belite Bio remains on track to report final topline results from the trial in the fourth quarter of 2025. These final results will be submitted to the NMPA as part of the NDA, which is being prepared in accordance with guidance from China’s Center for Drug Evaluation (CDE).

The Phase 3 DRAGON trial is a pivotal, randomized, double-masked, placebo-controlled, global study designed to evaluate the safety and efficacy of Tinlarebant in adolescent patients with Stargardt disease. The trial enrolled 104 participants across 11 jurisdictions, including the United States, United Kingdom, Germany, France, Belgium, Switzerland, Netherlands, China, Hong Kong, Taiwan, and Australia, with a 2:1 randomization of Tinlarebant to placebo. 

The primary efficacy endpoint is the growth rate of atrophic lesions, alongside evaluations of safety and tolerability. This acceptance of the NDA by the NMPA represents a significant step forward for Belite Bio and the Stargardt disease community, bringing the company closer to potentially providing the first approved treatment for this rare and debilitating retinal disorder.