Lin BioScience announced that the European Medicines Agency 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 1.3 in 10,000 births in the European Union. 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.
“Adding orphan designation in Europe to the recent orphan drug designation in the United States marks another important regulatory milestone in the development program for LBS-008,” Dr. Tom Lin, CEO of Lin Bioscience, said in a company news release. “We look forward to receiving our interim phase 1 clinical trial results later this year.”
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 the eye’s visual cycle, which is dependent on presence of vitamin A1 (retinol). LBS-008 works by reducing both the amount of circulating vitamin A1, as well as its uptake into the eye, thus 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 1 clinical trials. LBS-007 has also received orphan drug designation from the US Food & Drug Administration (FDA) in 2017.