Rare disease spotlight: Stargardt’s first approval in sight as next-gen aims to restore vison

Phase III data from Belite Bio has teed up the first drug approval for the rare, inherited childhood blindness Stargardt disease, offering early-stage patients a way to slow disease progression. The coming year is also poised to deliver clinical data on vision restoration strategies whose benefit could extend to later-stage patients, including those with few intact photoreceptors.

Over 900 gene variants have been reported to cause Stargardt disease, most commonly in the ABCA4 transporter, which helps clear by-products produced during the visual cycle — the process that continually refreshes the light-sensing pigments in the retina’s photoreceptor cells. Loss of ABCA4 function allows these by-products to build up to toxic levels, driving photoreceptor loss and progressive macular degeneration in children...