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APR 21 2027
UPCOMING · 264 DAYS TO PDUFA

Belite Bio tinlarebant $BLTE

for Stargardt disease type 1 (juvenile macular dystrophy)

NDA · Standard Review · Orphan · Breakthrough · Fast Track

Accepted JUN 12 2026.

About

Stargardt disease is an inherited retinal disorder that causes progressive macular degeneration and vision loss, often beginning in childhood. Tinlarebant is an oral drug that blocks retinol-binding protein 4 (RBP4), a key carrier of vitamin A to the eye. By limiting the supply of vitamin A to the retina, the drug may slow the toxic buildup of waste products that drive vision loss in these patients.

Pivotal trial

DRAGON Phase 3 completed NCT05244304

The primary objective of this trial is to assesses the efficacy of tinlarebant in slowing the rate of growth of atrophic lesion(s) in adolescent subjects with STGD1

Source: ClinicalTrials.gov

Also in development