Lin BioScience Receives US FDA Orphan Drug Status for LBS-008 for the Treatment of Stargardt Disease
Drug candidate targeting disease that causes of blindness in children scheduled to complete NIH Blueprint-funded Phase I clinical trials in 2018
SAN DIEGO--(BUSINESS WIRE)--Lin BioScience, a drug development company specializing in innovative therapies for oncology, ophthalmology and metabolic diseases, announced today that 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.
Our LBS-008 receives FDA Orphan Drug Status 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,” stated Dr. Tom Lin, CEO of Lin Bioscience. “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.
About Orphan Drug Designation
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.
About Lin BioScience, Inc.
Lin BioScience, Inc. is a drug development company specializing in innovative therapies for diseases with unmet medical needs. Lin BioScience's diverse pipeline consists of first-in-class drugs aimed at treating life-threatening or disabling diseases in ophthalmology, oncology and metabolic diseases.