Alkeus Pharmaceuticals has received the US Food and Drug Administration (FDA) Rare Pediatric Disease and Fast Track designations for its investigational oral therapy gildeuretinol (ALK-001).
Gildeuretinol is a chemically-modified vitamin A, formulated as a once-a-day oral therapy for the treatment of Stargardt Disease, and is currently in clinical development.
Stargardt is a rare and serious disease that causes severe vision impairment in children and young adults due to a defective ABCA4 protein and currently has no approved treatment.
The defect in the protein results in the advanced dimerisation of vitamin A, forming toxic by-products that irreversibly damage the retina, resulting in progressive vision loss.
According to the company, gildeuretinol is the first and only drug to address the underlying cause of Stargardt disease, by reducing the rate of vitamin A dimerisation in the eye.
Alkeus Pharmaceuticals president and CEO Michel Dahan said: “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.”
FDA grants Rare Paediatric Disease designation to the drugs intended to treat serious rare diseases that primarily affect individuals from birth to 18 years of age.
The designation provides Alkeus with a priority review voucher (PRV) upon approval that could be used to advance another clinical development programme.
FDA grants Fast Track designation to facilitate the development and speed up the review of treatments for a serious condition, with the potential to address an unmet medical need.
Gildeuretinol has previously received the FDA Breakthrough Therapy and Orphan Drug designations.
The drug was evaluated in the TEASE studies, which include four independent clinical studies of gildeuretinol, TEASE-1, TEASE-2, TEASE-3 and TEASE-4.
In the studies, gildeuretinol showed a favourable safety and tolerability profile, with no adverse events related to hyper- or hypo-vitaminosis A.
Kay added: “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.
“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.”
