Belite Bio Submits New Drug Application to FDA for its Stargardt Treatment

Belite Bio recently filed a new drug application with the U.S. Food and Drug Administration for tinlarebant, its oral drug to treat Stargardt disease, making it the first Stargardt treatment to make it this far in the FDA approval process. There are currently no treatments for Stargardt disease, a genetic eye disorder often diagnosed in childhood that causes central vision loss.

Stargardt is typically caused by two mutations in the ABCA4 gene, which cause toxic byproducts from the processing of vitamin A to build up and damage the retina, causing vision loss. Tinlarebant works by lowering levels of a protein that carries vitamin A from the liver to the eye, reducing build-up of vitamin A-based toxins in the eye. It is administered as a once-daily oral pill.

FDA has 60 days to complete its review. Tinlarebant has previously received fast-track and breakthrough therapy designations from FDA. This means that after reviewing the new drug application, FDA can prioritize tinlarebant’s review and could approve it for marketing within six months instead of the customary ten months.

The FDA application submission follows successful results from the drug’s phase 3 clinical trial reported in December 2025. Tinlarebant slowed down the growth of lesion area by 36 percent, compared to patients who received a placebo.

Belite Bio used data from the Foundation’s natural history study of patients with Stargardt disease to inform their clinical trial design. ProgStar, the largest ever natural history study of patients with Stargardt disease, followed 259 patients for two years, collecting imaging and medical record data to help clinicians and researchers understand how the disease progresses. The ProgStar study also created a database of Stargardt patients for future clinical trials, including 365 patients in 10 international clinical centers.

Belite Bio is also currently conducting other clinical trials for tinlarebant in Stargardt patients in the U.S., U.K., and Japan. Because Stargardt disease is sometimes called early onset macular degeneration, the treatment could also have applications in patients with an advanced form of dry age-related macular degeneration called geographic atrophy. A phase 3 clinical trial for using tinlarebant to treat geographic atrophy is ongoing.

Tinlarebant is one of several potential treatments designed for patients with an ABCA4-associated Stargardt diagnosis. See the Foundation’s Clinical Trial Pipeline for the latest on treatments making their way through clinical trials.