Belite Bio has announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to Tinlarebant, its investigational therapy for Stargardt disease (STGD1). This significant regulatory milestone is fueled by data from the ongoing global phase 3 DRAGON clinical trial, which focuses on Tinlarebant´s potential in addressing an urgent unmet medical need for patients with STGD1—a genetic eye disorder that currently lacks approved treatments.
The designation is based on a pre-specified interim analysis of the DRAGON trial, which is a randomized, double-masked, placebo-controlled, multi-jurisdictional study comprised of 104 adolescent patients. The Data Safety and Monitoring Board determined that Tinlarebant demonstrated substantial improvement over existing therapies on at least one clinically significant endpoint, fulfilling the criteria for Breakthrough Therapy status. Notably, early results indicate a favorable safety profile and stable visual acuity for most participants, with average vision changes of fewer than three letters over two years. The trial´s primary endpoint is the rate of growth of atrophic retinal lesions, along with comprehensive assessments of safety and tolerability.
Tinlarebant is an orally administered, once-daily tablet intended for early intervention to maintain retinal tissue health in both STGD1 and geographic atrophy (GA) patients. With no FDA-approved treatments for STGD1 currently available, Tinlarebant stands to become the first novel oral therapy for this population if approved. The global DRAGON trial spans the United States, United Kingdom, Germany, France, Belgium, Switzerland, Netherlands, China, Hong Kong, Taiwan, and Australia, and is expected to deliver its complete data readout by the end of the year. The advancement of Tinlarebant underscores growing momentum in genetic and inherited retinal disorder therapies, with the hope of addressing significant gaps in ophthalmic care.
