Lin BioScience announced the US Food and Drug Administration (FDA) has granted orphan drug designation to LBS-008, a first-in-class oral therapy for the treatment of Stargardt Disease.
Stargardt Disease is currently an untreatable inherited condition that causes permanent vision loss in children during childhood and adolescence. It is the most common form of juvenile macular degeneration with an incidence of approximately 1 in 10,000 births. The disease is caused by a mutation in the ABCA4 gene, which leads to the accelerated formation and accumulation of toxic vitamin A dimers in the retina that cause progressive retinal cell death and permanent loss of vision.
“Receiving orphan drug status is an important regulatory milestone in the development of our lead candidate, and highlights the substantial unmet medical need for new therapies to address Stargardt Disease,” Dr. Tom Lin, CEO of Lin Bioscience said. “Our team, with the support of the NIH Blueprint Program, is driving LBS-008 through Phase I clinical trials in order to help patients suffering from this devastating, untreatable condition.”
LBS-008 is a first-in-class oral therapy that prevents the buildup of toxins in the eye that cause Stargardt Disease and atrophic Age-related Macular Degeneration (dry AMD). The toxins are a by-product of the eye’s visual cycle, which is dependent on presence of vitamin A1 (retinol). LBS-008 works by reducing both the amount of circulating retinol binding protein (RBP4) and retinol, thus reducing its excess uptake into the eye and preventing the formation of the toxins associated with macular degeneration. The US National Institute of Health's (NIH) Blueprint Neurotherapeutics Network, which has funded the therapy’s discovery and development, will continue to provide support and funding through to the completion of Phase I clinical trials.
The US FDA orphan drug designation provides incentives for companies to develop drugs for rare diseases affecting fewer than 200,000 patients. These incentives may include FDA assistance in clinical trial design, tax credits towards the cost of clinical trials, prescription drug user fee waivers, and potential market exclusivity for seven years following approval.