FDA Grants Breakthrough Status to Tinlarebant for Stargardt Disease

FDA Grants Breakthrough Status to Tinlarebant for Stargardt Disease

By, Admin 24 May 2025

Belite Bio, Inc., a clinical-stage biopharmaceutical company focused on treating degenerative retinal diseases, announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for its investigational drug Tinlarebant for Stargardt disease type 1 (STGD1). The decision is based on interim data from the ongoing Phase 3 DRAGON trial.
There are currently no approved treatments for STGD1, a progressive and inherited eye condition that often leads to legal blindness.

A Milestone for Stargardt Patients

Dr. Tom Lin, Chairman and CEO of Belite Bio, stated:

“Breakthrough Therapy Designation is an exciting milestone that underscores Tinlarebant’s potential to address a serious unmet need for patients with STGD1.""
He added that the company is fully committed to the Stargardt community and anticipates data from the DRAGON trial by the end of this year.

What Is Breakthrough Therapy Designation?

The FDA’s Breakthrough Therapy Designation is granted to drugs that show:

  • Preliminary clinical evidence of substantial improvement over existing therapies
  • Promising effects on one or more clinically significant endpoints
  • Potential to expedite the development and regulatory review process

Benefits include:

  • Rolling submission of regulatory documents
  • Priority review
  • More frequent communication with FDA officials to ensure timely development

DRAGON Trial Overview

The Phase 3 DRAGON trial is a pivotal, randomized, double-masked, placebo-controlled global study, which includes:

  • 104 adolescent STGD1 patients
  • 11 countries and regions: United States, United Kingdom, Germany, France, Belgium, Switzerland, Netherlands, China, Hong Kong, Taiwan, and Australia
  • 2:1 randomization (Tinlarebant vs. placebo)
  • Primary endpoint: Growth rate of atrophic retinal lesions
  • Additional assessments: Safety and tolerability

Promising Interim Data

The Breakthrough Therapy Designation was supported by pre-specified interim analysis, which showed:

  • Stable visual acuity: Mean change of less than three letters over two years
  • Favorable safety profile, consistent with prior data and Tinlarebant’s mechanism of action

Scientific Insights and Development Background

Dr. Nathan L. Mata, Chief Scientific Officer of Belite Bio, highlighted the innovation behind Tinlarebant:

“This designation validates our approach of targeting the root cause of STGD1 by inhibiting retinol binding protein 4 (RBP4).”

Key scientific insights:

  • STGD1 is driven by toxic byproducts of vitamin A metabolism
  • Tinlarebant works by modulating RBP4 to reduce these toxic accumulations
  • Development is based on more than two decades of preclinical research

Unmet Needs and Future Potential

Dr. Hendrik Scholl, Chief Medical Officer at Belite Bio, emphasized the urgency:

“STGD1 begins in adolescence and often results in legal blindness. Tinlarebant offers new hope to patients with no treatment options.”

Key facts:

  • STGD1 significantly impacts quality of life
  • Tinlarebant is being developed as a once-daily oral tablet
  • It may also be applicable for Geographic Atrophy (GA) in age-related macular degeneration
  • If approved, it would be the first oral treatment for both STGD1 and GA

About Belite Bio

Belite Bio is dedicated to developing novel therapies for retinal degenerative diseases and metabolic disorders. Its lead programs include treatments for:

  • Stargardt disease type 1 (STGD1)
  • Geographic Atrophy (GA) in dry age-related macular degeneration
  • Other targeted metabolic diseases

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