Key Takeaways

  • The FDA has accepted Belite Bio’s NDA for tinlarebant in Stargardt disease type 1 and granted Priority Review, with a PDUFA date of February 12, 2027
  • The phase 3 DRAGON trial showed a 35.7% reduction in the growth rate of atrophic retinal lesions with tinlarebant compared with placebo
  • If approved, tinlarebant would become the first FDA-approved treatment for Stargardt disease type 1

The FDA has accepted Belite Bio’s new drug application (NDA) for tinlarebant for the treatment of Stargardt disease type 1 (STGD1) and granted the application Priority Review, the company announced.

The agency has set a Prescription Drug User Fee Act (PDUFA) target action date of February 12, 2027. If approved, tinlarebant would become the first FDA-approved treatment for STGD1.

The NDA is supported by results from the phase 3 DRAGON trial, which evaluated tinlarebant in patients with STGD1. According to the company, treatment with tinlarebant resulted in a statistically significant 35.7% reduction in the growth rate of atrophic retinal lesions compared with placebo. Lesion growth was assessed as definitely decreased autofluorescence (DDAF) using fundus autofluorescence imaging.

Belite Bio reported that tinlarebant has been generally well tolerated in clinical trials, with adverse effects consistent with its mechanism of action.

“The acceptance of our NDA with Priority Review designation underscores the immediate need among the Stargardt disease community for an approved treatment option, and we believe it reinforces the quality and depth of the data that we’ve generated,” said Tom Lin, MD, chairman and CEO of Belite Bio.

Dr. Lin added that the company plans to work with the FDA during the review while continuing preparations for a potential commercial launch.

STGD1 is a rare inherited retinal disorder associated with mutations in the ABCA4 gene. The condition typically causes progressive retinal degeneration and irreversible vision loss, often beginning early in life. Belite Bio estimates that approximately 53,000 people in the United States are affected.

There are currently no FDA-approved pharmacologic treatments for STGD1, leaving patients reliant primarily on supportive measures, including low-vision aids.

Tinlarebant is an oral, once-daily investigational therapy being developed for degenerative retinal diseases. With Priority Review, the FDA aims to take action on applications for therapies that could offer significant improvements in the treatment, diagnosis, or prevention of serious conditions.