Belite Bio announces NDA acceptance of tinlarbant by China’s NMPA

Belite Bio recently announced the acceptance of its New Drug Application (NDA) with priority review for tinlarebant by the Center for Drug Evaluation of China’s National Medical Products Administration (NMPA).¹ The phase 3 DRAGON study (NCT05244304), investigating the use of tinlarebant for the treatment of Stargardt disease type 1 (STGD1), informed this acceptance.

“Having China NMPA agreed to review the NDA based on interim phase 3 data is a remarkable milestone for Belite Bio and the Stargardt community,” Tom Lin, MD, PhD, chairman and CEO of Belite Bio, said in a press release.¹

“This milestone underscores the strength of the program and the urgent need for therapies in this devastating disease, where no approved treatment options exist. This achievement positions Belite Bio to advance tinlarebant through the final stages of development and, if successful, bring the first treatment to people living with Stargardt disease.”

In September of this year, Belite Bio concluded the phase 3 study, which enrolled 104 participants aged 12 to 20 years old from 11 different regions, including Australia, Belgium, the US, the UK, Germany, France, Switzerland, China, the Netherlands, Hong Kong, and Taiwan.² The patients were randomized, with a 2:1 randomization (tinlarebant:placebo).¹

According to the company, the interim analysis results of DRAGON showed statistical significance in the primary endpoint, which is defined by the company as “the growth rate of atrophic lesions, alongside the assessment of safety and tolerability.”¹

A once-a-day oral medication, tinlarebant aims to decrease the buildup of vitamin A-based toxins known as bisretinoids.^2,3

“With no approved therapies available today, tinlarebant has the potential to be the first treatment for this devastating inherited macular degeneration,” Lin said in a press release earlier this year.²

“We are deeply grateful to the patients, families, investigators, and study teams worldwide who made this clinical trial possible.”

In addition to the recent acceptance of the NDA with priority review from China's NMPA, tinlarebant has previously received fast-track designation and rare pediatric disease designation in the US; orphan drug designation in the US, Japan, and Europe; and the Sakigake designation in Japan for the treatment of STGD1.^1,2

Reference:

1. Belite Bio Announces China NMPA Agrees to New Drug Application with Priority Review based on Interim Analysis Results for the Treatment of Stargardt Disease with Tinlarebant. BioSpace. Published October 16, 2025. Accessed October 21, 2025. https://www.biospace.com/press-releases/belite-bio-announces-china-nmpa-agrees-to-new-drug-application-with-priority-review-based-on-interim-analysis-results-for-the-treatment-of-stargardt-disease-with-tinlarebant

2. Filkins K. Belite Bio concludes phase 3 DRAGON study for Stargardt disease. Ophthalmologytimes.com. Published September 16, 2025. https://www.ophthalmologytimes.com/view/belite-bio-concludes-phase-3-dragon-study-for-stargardt-disease

3. Filkins K. FDA grants Breakthrough Therapy Designation for Tinlarebant. Ophthalmologytimes.com. Published May 21, 2025. https://www.ophthalmologytimes.com/view/fda-grants-breakthrough-therapy-designation-for-tinlarebant