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Belite Bio Announces U.S. Food and Drug Administration Acceptance and Priority Review of New Drug Application for Tinlarebant for the Treatment of Stargardt Disease Type 1

BLTE
  • The FDA accepted the New Drug Application for tinlarebant & granted Priority Review designation.
  • The Prescription Drug User Fee Act (PDUFA) target action date is set for February 12, 2027.
  • If approved, tinlarebant would be the first-ever FDA-approved treatment for Stargardt Disease Type 1 (STGD1).
  • The filing was based on Phase 3 DRAGON trial results showing a 35.7% reduction in atrophic retinal lesion growth versus placebo.
  • Tinlarebant has demonstrated a generally well-tolerated safety profile with side effects consistent with its mechanism of action.
  • STGD1 affects an estimated 53,000 people in the U.S. & is caused by ABCA4 gene mutations leading to progressive vision loss.
  • Tinlarebant works by reducing serum retinol binding protein 4 (RBP4) to lower bisretinoid toxin accumulation in the eye.
  • The drug holds multiple regulatory designations including Breakthrough Therapy, Fast Track, Orphan Drug, & Rare Pediatric Disease status.
  • Belite Bio is also evaluating tinlarebant in Phase 2/3 DRAGON II trials for STGD1 & Phase 3 PHOENIX trials for geographic atrophy.