Belite Bio has received Breakthrough Therapy Designation from the FDA for Tinlarebant, its treatment for Stargardt disease (STGD1), based on positive interim Phase 3 DRAGON trial data. This designation is significant because there are no currently approved treatments for STGD1, a genetic eye disease leading to progressive vision loss starting in adolescence. The FDA’s decision was based on evidence suggesting Tinlarebant offers substantial improvement over existing options, supported by the drug’s favorable safety profile and its ability to maintain visual acuity in most trial participants over two years.
This breakthrough designation holds considerable promise for young patients facing a future of declining vision due to STGD1. The designation acknowledges the potential for Tinlarebant to significantly alter the disease’s progression, offering a much-needed intervention where none currently exist. The demonstrated stability in visual acuity over a two-year period is particularly encouraging, highlighting the potential to preserve vision and improve the quality of life for those affected by STGD1.
The Phase 3 DRAGON trial, a global study involving 104 participants across 11 jurisdictions, is evaluating Tinlarebant’s safety and efficacy in adolescent STGD1 patients. The primary efficacy endpoint is the growth rate of atrophic lesions. Tinlarebant, an oral once-daily tablet, targets the underlying disease mechanism by reducing the accumulation of toxic byproducts of vitamin A in the retina. The Breakthrough Therapy Designation expedites the drug’s development and review process, including eligibility for rolling review and priority review.
This designation represents a pivotal step towards potential approval for Tinlarebant and offers a significant advancement in the field of inherited retinal diseases. The expedited development and review process facilitated by the breakthrough designation increases the likelihood of bringing this potentially transformative therapy to patients sooner. The final results of the DRAGON trial, expected by Q4 2025, will be crucial in determining the next steps for Tinlarebant and shaping the future treatment landscape for STGD1.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
