Gildeuretinol receives FDA rare pediatric disease and fast track designations for Stargardt disease treatment

Alkeus Pharmaceuticals, Inc, announced that its investigational oral therapy, gildeuretinol (ALK-001), has received Rare Pediatric Disease and Fast Track designations from the U.S. Food and Drug Administration (FDA) for treating Stargardt disease. It has previously been granted Breakthrough Therapy and Orphan Drug designations.

At the 2024 American Academy of Ophthalmology meeting, data from Alkeus’ TEASE clinical program highlighted promising results. The TEASE-1 study, a 2-year, placebo-controlled trial, showed that gildeuretinol reduced the growth rate of retinal lesions by 21.6% compared to untreated patients (P < 0.001). A separate analysis demonstrated a 29.5% reduction. In addition, interim data from TEASE-3 suggested that early-stage Stargardt patients treated with gildeuretinol remained asymptomatic and had stable visual acuity over up to six years.

Gildeuretinol has demonstrated a favorable safety profile, with no treatment-related adverse events such as night blindness or delays in dark adaptation.

Read the full press release here.