Belite Bio Initiates Rolling NDA Submission for Tinlarebant to Treat Stargardt Disease

Belite Bio has initiated a rolling submission of a New Drug Application to the U.S. Food and Drug Administration for tinlarebant, an investigational oral therapy for Stargardt disease type 1, a rare inherited retinal disorder. Tinlarebant has Breakthrough Therapy Designation from the FDA, and the company expects to complete the rolling submission in the second quarter of 2026.

The initiation marks an important milestone, as tinlarebant could become the first approved treatment for Stargardt disease. "Stargardt disease is a devastating, progressive disease that typically begins in adolescence and leads to legal blindness in virtually all cases," said the Chief Medical Officer of Belite Bio. "I am proud of the data package that we are submitting to the FDA, including the transformative results from our Phase 3 DRAGON trial, and look forward to completing the NDA submission in the second quarter of this year."

The Chairman and CEO of Belite Bio expressed gratitude to patients, caregivers, and investigators, and noted that the company has been working toward this milestone since releasing pivotal trial data demonstrating tinlarebant's impact on slowing retinal degeneration. "We are dedicated to bringing this potential treatment to patients in the U.S. and worldwide, and we are focused on continuing our commercialization preparation work, building out key teams including sales, market access, medical affairs, marketing, regulatory, and operations to ensure a seamless potential launch next year."

Tinlarebant is a novel oral therapy designed to reduce the accumulation of vitamin A-based toxins, known as bisretinoids, that cause retinal disease in STGD1. It works by reducing and maintaining levels of serum retinol binding protein 4, the sole carrier protein for retinol transport from the liver to the eye. The drug has been granted Breakthrough Therapy Designation, Fast Track Designation, and Rare Pediatric Disease Designation in the U.S., Orphan Drug Designation in the U.S., Europe, and Japan, and Sakigake Designation in Japan.

Belite Bio is a clinical-stage biopharmaceutical company focused on degenerative retinal diseases. Its lead candidate, tinlarebant, has completed a Phase 3 trial in adolescent STGD1 subjects and is currently being evaluated in a Phase 2/3 trial in adolescent STGD1 subjects and a Phase 3 trial in subjects with geographic atrophy.