for Stargardt disease type 1 (juvenile macular dystrophy)
Sponsor announced submission APR 21 2026. FDA filing review (~60 days) in progress; PDUFA date estimated as acceptance + 10 months Standard Review. Card updates with confirmed dates when FDA acceptance announcement lands.
Stargardt disease is an inherited retinal disorder that causes progressive vision loss, often starting in childhood or adolescence. Tinlarebant is an oral drug that blocks retinol-binding protein 4 (RBP4), a protein involved in transporting vitamin A to the retina. By reducing vitamin A buildup in the eye, tinlarebant may slow the retinal damage that leads to vision loss in patients with Stargardt disease.
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