Belite Bio (BLTE) FDA Approvals

Belite Bio's Drug in the FDA Approval Process

This section highlights FDA-related milestones and regulatory updates for drugs developed by Belite Bio (BLTE). Over the past two years, Belite Bio has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as Tinlarebant. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.

Tinlarebant FDA Regulatory Timeline and Events

Tinlarebant is a drug developed by Belite Bio for the following indication: In Stargardt Disease. This drug is under review by the U.S. Food and Drug Administration (FDA). Below is a timeline of key regulatory milestones for this therapy.

Oral presentation - May 15,2026

• Drug: Tinlarebant
• Announced Date: May 15, 2026
• Indication: In Stargardt Disease

Belite Bio, Inc. announced that the Company will give an oral presentation at the 27th Fundus Disease Forum and International Retinal Symposium (Retina China 2026) being held from May 21-23, 2026, in Nanjing, China.

Belite Bio, Inc. said it will give an oral presentation at the 27th Fundus Disease Forum and International Retinal Symposium (Retina China 2026), taking place May 21-23, 2026, in Nanjing, China. The presentation will highlight previously reported positive topline results from the Phase 3 DRAGON trial, which studied tinlarebant as a treatment for Stargardt disease type 1 (STGD1).

In the trial, tinlarebant showed a 35.7% reduction in the growth rate of atrophic retinal lesions compared with placebo. The company also said the drug was generally well tolerated, with side effects that matched what is expected from its mechanism of action. These results support Belite Bio’s ongoing work on tinlarebant as a possible treatment for this rare eye disease.

Oral presentation - April 27,2026

• Drug: Tinlarebant
• Announced Date: April 27, 2026
• Indication: In Stargardt Disease

Belite Bio, Inc announced that the Company will give an oral presentation at the Retinal Therapeutics Innovation Summit being held on May 1, 2026, in Denver, Colorado.

Belite Bio, Inc. announced it will give an oral presentation at the Retinal Therapeutics Innovation Summit on May 1, 2026, in Denver, Colorado.

The presentation will cover the previously disclosed positive topline results from the Phase 3 DRAGON trial of tinlarebant for treating Stargardt disease type 1 (STGD1). In the study, tinlarebant produced a clinically meaningful 35.7% reduction in the growth rate of atrophic retinal lesions versus placebo and showed a strong safety profile. A reduction of this size could help slow vision loss for people with STGD1.

Company executives and study investigators will present detailed data, explain the clinical meaning of the results, and discuss implications for patients and ongoing clinical development. The session gives clinicians, researchers, and investors a chance to learn more about tinlarebant’s effects and safety.

The presentation will be followed by a brief question-and-answer session. For media and investor inquiries: Jennifer Wu [email protected] and Julie Fallon [email protected].

Enrollment Completion - January 27,2026

Phase 2/3

• Drug: Tinlarebant
• Announced Date: January 27, 2026
• Indication: In Stargardt Disease

Belite Bio, Inc nnounced the completion of enrollment of 60 subjects, including 15 Japanese subjects, in the Phase 2/3 DRAGON II clinical trial evaluating tinlarebant for the treatment of Stargardt disease type 1 (STGD1).

Belite Bio announced completion of enrollment of 60 adolescent subjects, including 15 Japanese participants, in the Phase 2/3 DRAGON II clinical trial of tinlarebant for Stargardt disease type 1 (STGD1). DRAGON II is a global, 24‑month, randomized, double‑masked, placebo‑controlled study that enrolled 12–20‑year‑old patients across Japan, the United States, and the United Kingdom. Participants were randomized 1:1 to receive tinlarebant or placebo, meeting the study’s targeted sample size.

The company thanked investigators, the adolescent participants, and their families for supporting the trial. Belite’s leadership noted that completing enrollment advances their clinical development program and that the study was implemented to align with requirements of the Japanese Pharmaceuticals and Medical Devices Agency, supporting potential regulatory paths in Japan while gathering data for adolescents globally.

Tinlarebant is an oral therapy designed to lower retinol transport to the eye and reduce formation of toxic bisretinoids. DRAGON II will evaluate its efficacy, safety, and tolerability in adolescent STGD1 patients.

Positive Results - December 1,2025

Phase 3

• Drug: Tinlarebant
• Announced Date: December 1, 2025
• Indication: In Stargardt Disease

Belite Bio, Inc announced topline results from the global Phase 3 "DRAGON" trial of Tinlarebant, marking the first successful pivotal trial in patients with Stargardt disease type 1 (STGD1).

Belite Bio reported topline results from the global Phase 3 DRAGON trial showing Tinlarebant is the first therapeutic to demonstrate clinical efficacy in Stargardt disease type 1 (STGD1). In 104 adolescent patients (age 12–20) treated over 24 months, Tinlarebant reduced the growth rate of retinal atrophic lesions (measured as definitely decreased autofluorescence, DDAF, by fundus autofluorescence imaging) by about 36% versus placebo. The primary analysis reached statistical significance (p = 0.0033), and a post-hoc longitudinal analysis showed an even stronger signal (p < 0.0001).

Tinlarebant was generally well tolerated, with four treatment-related discontinuations and mostly mild ocular effects such as xanthopsia and delayed dark adaptation; visual acuity changed little over 24 months, consistent with disease course. Belite plans to pursue regulatory filing and will share more data at upcoming medical meetings.

Regulatory Update - October 15,2025

NDA

• Drug: Tinlarebant
• Announced Date: October 15, 2025
• Indication: In Stargardt Disease

Belite Bio, Inc. announced that the Center for Drug Evaluation of China's National Medical Products Administration ("NMPA") has agreed to accept the New Drug Application (NDA) with priority review for Tinlarebant for the treatment of Stargardt disease based on the interim analysis results from the Phase 3 DRAGON trial.

Belite Bio, Inc. announced on October 15, 2025, that China’s National Medical Products Administration (NMPA) has agreed to accept its New Drug Application (NDA) for priority review of Tinlarebant to treat Stargardt disease. This decision is based on interim analysis results from the Phase 3 DRAGON trial.

The interim analysis demonstrated statistical significance in the trial’s primary endpoint, measuring the growth rate of atrophic eye lesions. The randomized, double-masked, placebo-controlled study enrolled 104 adolescent subjects across 11 jurisdictions with a 2:1 Tinlarebant-to-placebo ratio. Final topline data are expected in the fourth quarter of 2025 to support the full NDA submission.

Tinlarebant is an oral therapy that lowers retinol binding protein 4 to reduce vitamin A–based toxins in the eye. Priority review by the NMPA is a major milestone for Belite Bio and the Stargardt community, which has no approved treatments. If approved, Tinlarebant could become the first therapy for this degenerative eye disease.

Provided Update - September 12,2025

Phase 3

• Drug: Tinlarebant
• Announced Date: September 12, 2025
• Indication: In Stargardt Disease

Belite Bio Inc announced the completion of the last subject visit in the Phase 3 DRAGON clinical trial evaluating Tinlarebant for the treatment of Stargardt disease type 1 (STGD1).

Belite Bio Inc announced the final subject visit was completed in its pivotal Phase 3 DRAGON trial of Tinlarebant for treating Stargardt disease type 1 (STGD1). Dr. Tom Lin, Chairman and CEO, said this milestone brings the company closer to offering the first approved therapy for this inherited macular degeneration and thanked patients, families, investigators, and study teams worldwide.

The DRAGON trial enrolled 104 adolescent subjects across 11 countries, including the U.S., U.K., Germany, France, Belgium, Switzerland, the Netherlands, China, Hong Kong, Taiwan, and Australia, with a 2:1 randomization of Tinlarebant versus placebo. Ninety-four subjects completed the study, with the last visit on September 11, 2025. The primary endpoint is the growth rate of atrophic lesions, and safety and tolerability of Tinlarebant will also be evaluated.

Belite Bio expects to report topline results from the DRAGON trial in the fourth quarter of 2025 and plans to file New Drug Applications in the first half of 2026.

Enrollment Update - July 2,2025

Phase 3

• Drug: Tinlarebant
• Announced Date: July 2, 2025
• Indication: In Stargardt Disease

Belite Bio, Inc announced the completion of enrollment in the PHOENIX trial, a global, 24-month Phase 3 pivotal trial evaluating the safety and tolerability of Tinlarebant and its potential to reduce atrophic lesion growth rate in patients diagnosed with geographic atrophy (GA) in dry age-related macular degeneration (AMD).

Belite Bio, Inc. announced that enrollment is now complete for its PHOENIX trial, a global, 24-month Phase 3 study. The trial will examine the safety and tolerability of Tinlarebant, an investigational oral treatment, and its ability to slow the growth of atrophic lesions in patients with geographic atrophy (GA) due to dry age-related macular degeneration (AMD).

This pivotal study enrolled 500 subjects from the United States, United Kingdom, France, Czech Republic, Switzerland, China, Taiwan, and Australia. The completion of enrollment marks a key milestone in the development of Tinlarebant, which could offer hope for patients suffering from this serious, progressive eye condition that currently has no approved oral treatment. Researchers look forward to sharing the trial’s interim results and further insights into how Tinlarebant may help slow the progression of GA.

Designation Grant - May 21,2025

Breakthrough Therapy

• Drug: Tinlarebant
• Announced Date: May 21, 2025
• Indication: In Stargardt Disease

Belite Bio, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for Tinlarebant for the treatment of Stargardt disease (STGD1) based on the previously reported interim data from the ongoing Phase 3 DRAGON trial.

Belite Bio, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for Tinlarebant to treat Stargardt disease (STGD1). This designation was given based on promising interim results from the ongoing Phase 3 DRAGON trial. The trial’s results show that Tinlarebant has a strong safety profile and may help stabilize visual acuity in most adolescent patients with the condition. The drug’s potential to slow or improve the disease process marks a major milestone for patients who currently have no approved treatment options for STGD1.

The Breakthrough Therapy Designation supports an accelerated development and review process. With the DRAGON trial expected to complete by the fourth quarter of 2025, Belite Bio continues to work hard to bring a new, effective therapy to those affected by this serious, progressive eye disease.

Analysis - February 27,2025

Phase 3

• Drug: Tinlarebant
• Announced Date: February 27, 2025
• Indication: In Stargardt Disease

Belite Bio, Inc announced that following a pre-specified Interim Analysis of the pivotal global Phase 3 "DRAGON" trial data of Tinlarebant in adolescent Stargardt disease patients, the Data Safety Monitoring Board (DSMB) has recommended the trial proceed without any modifications.

Belite Bio, Inc. announced an important milestone in its Phase 3 DRAGON trial for Tinlarebant in adolescent Stargardt disease patients. After a pre-specified interim analysis, the independent Data Safety Monitoring Board (DSMB) recommended that the trial proceed without any modifications. This means the current study design and sample size of 104 subjects will remain unchanged, as the data did not suggest a need for an increase.

The analysis was conducted after one year of assessments, showing that Tinlarebant is well-tolerated and that the visual acuity in most patients remained stable. The DSMB also advised that the current data be submitted for further regulatory review. The trial is on track to be completed by the fourth quarter of 2025, including a brief follow-up period after the final assessments.

Dose Update - September 10,2024

Phase 2/3

• Drug: Tinlarebant
• Announced Date: September 10, 2024
• Indication: In Stargardt Disease

Belite Bio, Inc announced that the first patient has been dosed at the Tokyo Medical Center in the Phase 2/3 portion of its DRAGON II clinical trial evaluating Tinlarebant for the treatment of STGD1.

Belite Bio, Inc announced an important milestone in its fight against Stargardt Disease (STGD1). The first patient has been dosed at the Tokyo Medical Center as part of the Phase 2/3 portion of the DRAGON II clinical trial. This trial is designed to assess the safety, efficacy, and tolerability of Tinlarebant, an innovative oral therapy aimed at reducing harmful vitamin A-based toxins that contribute to retinal damage in STGD1 patients. Approximately 60 adolescent subjects will take part in the study across the U.S., U.K., and Japan. The trial follows a successful Phase 1b study in Japanese patients and marks a significant step toward developing a long-needed treatment for this degenerative retinal disease. Researchers and clinicians are hopeful that Tinlarebant can offer a new option for patients suffering from Stargardt Disease.

Designation Grant - June 12,2024

• Drug: Tinlarebant
• Announced Date: June 12, 2024
• Indication: In Stargardt Disease

Belite Bio announces that its lead pipeline, Tinlarebant, has been granted Sakigake Designation by the Ministry of Health, Labour and Welfare in Japan (MHLW) for the treatment of STGD1.

Belite Bio has received an important boost as its lead drug candidate, Tinlarebant, has been granted Sakigake Designation by Japan’s Ministry of Health, Labour and Welfare (MHLW) for treating Stargardt Disease (STGD1). This designation is given to innovative drugs with promising effectiveness that target serious diseases. With Sakigake, Tinlarebant will benefit from prioritized consultations, a focused pre-application review, and an accelerated review process, all of which may speed up its approval in Japan.

Tinlarebant is an oral tablet designed to slow the progression of STGD1 by reducing the buildup of harmful vitamin A-derived toxins in the eye. This recognition highlights the drug’s potential to address the unmet needs of patients with this inherited retinal disorder and may bring faster access to treatment for those affected.