for Stargardt disease
Sponsor announced submission . 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 caused by ABCA4 gene mutations that progressively destroys central vision, often starting in childhood. Tinlarebant is an oral drug that blocks retinol-binding protein, reducing toxic vitamin A byproducts that accumulate in the retina. By slowing this buildup, the treatment may help preserve remaining vision in adolescents with the most common form of the disease (STGD1).
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