Alkeus Pharmaceuticals has 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). These designations are intended to facilitate the development and expedite the review of gildeuretinol for the treatment of Stargardt disease, a rare and serious condition causing severe vision impairment in children and young adults.
Gildeuretinol is a chemically-modified vitamin A, formulated for once-daily oral administration. It is currently in clinical development as a potential treatment for Stargardt disease, which is caused by a defective ABCA4 protein. This defect leads to the excessive dimerization of vitamin A, forming toxic by-products that irreversibly damage the retina, resulting in progressive vision loss. There are currently no approved treatments for Stargardt disease, highlighting a significant unmet medical need.
Addressing the Root Cause of Stargardt Disease
According to Alkeus Pharmaceuticals, gildeuretinol is the first and only drug designed to address the underlying cause of Stargardt disease by reducing the rate of vitamin A dimerization in the eye. By targeting the source of retinal damage, gildeuretinol aims to slow or halt the progression of vision loss in affected individuals.
Michel Dahan, President and CEO of Alkeus Pharmaceuticals, stated, "Receiving both the FDA’s Rare Paediatric Disease and Fast Track designations are important milestones for Alkeus that highlight the potential for oral gildeuretinol to be a groundbreaking therapy for patients. These designations were granted on top of the previously awarded Breakthrough Therapy and Orphan Drug designations. Together, these achievements recognise the significant unmet medical need in Stargardt disease and the overwhelming burden on patients as well as their families and caregivers."
Regulatory and Clinical Milestones
The FDA grants Rare Pediatric Disease designation to drugs intended to treat serious rare diseases primarily affecting individuals from birth to 18 years of age. This designation provides Alkeus with a priority review voucher (PRV) upon approval, which can be used to expedite the review of another clinical development program. The Fast Track designation is granted to treatments for serious conditions that have the potential to address unmet medical needs.
Gildeuretinol has been evaluated in the TEASE studies, comprising four independent clinical studies (TEASE-1, TEASE-2, TEASE-3, and TEASE-4). These studies have demonstrated a favorable safety and tolerability profile for gildeuretinol, with no adverse events related to hyper- or hypo-vitaminosis A.
Clinical Trial Data
Data from the TEASE studies suggest the potential value of early intervention with gildeuretinol in patients with Stargardt disease, even before the onset of progressive central vision loss. "TEASE-1 is the first randomised, controlled trial in Stargardt disease that has shown an efficacy endpoint, which is very exciting as an inherited retinal disease specialist taking care of patients with this devastating condition," said Kay, . "In addition, the TEASE-3 data indicate the potential value of treating patients with Stargardt disease as early as possible, before the onset of progressive central vision loss."
