FDA Events for Belite Bio (BLTE)
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.
- Drug:
- Tinlarebant
- Announced Date:
- July 2, 2025
- Indication:
- In Stargardt Disease
Announcement
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).
AI Summary
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.
Read Announcement- Drug:
- Tinlarebant
- Announced Date:
- May 21, 2025
- Indication:
- In Stargardt Disease
Announcement
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.
AI Summary
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.
Read Announcement- Drug:
- Tinlarebant
- Announced Date:
- February 27, 2025
- Indication:
- In Stargardt Disease
Announcement
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.
AI Summary
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.
Read Announcement- Drug:
- Tinlarebant
- Announced Date:
- September 10, 2024
- Indication:
- In Stargardt Disease
Announcement
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.
AI Summary
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.
Read Announcement- Drug:
- Tinlarebant
- Announced Date:
- June 12, 2024
- Indication:
- In Stargardt Disease
Announcement
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.
AI Summary
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.
Read Announcement - Drug:
- Tinlarebant
- Announced Date:
- May 6, 2024
- Indication:
- In Stargardt Disease
Announcement
Belite Bio, Inc announced additional findings from the 24-month Phase 2 study of Tinlarebant in adolescent Stargardt disease (STGD1) at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting.
AI Summary
Belite Bio, Inc. presented new data at the ARVO Annual Meeting from a 24‐month Phase 2 study of Tinlarebant in adolescent Stargardt disease (STGD1). The study analyzed visual acuity loss, retinal imaging, and genotypic profiles, showing that 42% of patients with pathogenic ABCA4 mutations did not develop new atrophic retinal lesions during treatment. Additionally, a novel lesion quantification method revealed that atrophic lesions in the macula halted their growth after 16 months, suggesting improved protection for retinal cells.
Notably, patients with a history of rapid vision loss experienced a significant reduction in the decline of their best-corrected visual acuity during the trial. These results support Tinlarebant’s potential to slow disease progression in STGD1 and offer a promising treatment approach for preserving retinal function and vision in affected adolescents.
Read Announcement
Belite Bio FDA Events - Frequently Asked Questions
As of now, Belite Bio (BLTE) has not received any FDA approvals for its therapy in the last two years.
In the past two years, Belite Bio (BLTE) has reported FDA regulatory activity for Tinlarebant.
The most recent FDA-related event for Belite Bio occurred on July 2, 2025, involving Tinlarebant. The update was categorized as "Enrollment Update," with the company reporting: "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)."
Currently, Belite Bio has one therapy (Tinlarebant) targeting the following condition: In Stargardt Disease.
More FDA Event Resources from MarketBeat
FDA Event Stage Terminology & Abbreviation Guide
- NDA: New Drug Application
- ANDA: Abbreviated New Drug Application
- sNDA: Supplemental New Drug Application
- BLA: Biologics License Application
- sBLA: Supplemental Biologics License Application
- FDA Approved: Approved by the FDA
- EMA: European Medicines Agency
- CE Mark: European Union Certification
- NMPA: China National Medical Products Administration
- MHLW: Japanese Ministry of Health
- FDA Meeting: Consultation with FDA
- Pre-IND: Pre-Investigational New Drug Meeting
- Breakthrough Therapy: Special FDA designation for promising therapies
- Fast Track: Accelerated FDA approval pathway
- Orphan Drug: Designation for rare disease treatments
- RPD: Rare Pediatric Disease Designation
- RMAT: Regenerative Medicine Advanced Therapy
- DSMB Review: Data Safety Monitoring Board Review
- IDMC Review: Independent Data Monitoring Committee
- MAA: MHRA Marketing Authorization Application
- RTF: Refusal to File (Rejected Application)
- 510(k): FDA Clearance for Medical Devices
- Rolling Submission: Staggered regulatory review process
This page (NASDAQ:BLTE) was last updated on 7/9/2025 by MarketBeat.com Staff
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