Lin BioScience, a drug development company targeting untreatable conditions in oncology, ophthalmology and metabolic diseases, announced today 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," stated Dr. Tom Lin, CEO of Lin Bioscience. "We look forward to receiving our interim Phase I 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 I clinical trials. LBS-007 has also received orphan drug designation from the US Food & Drug Administration (FDA) in 2017.
About EMA Orphan Drug Designation (ODD)
The European Medicines Agency (EMA) Orphan Drug Designation provides incentives for companies to develop drugs for rare diseases with a prevalence in the European Union (EU) not more than five in 10,000. These incentives may include protocol assistance, access to the EU centralized authorization procedure, reduced regulatory filing fees and ten years of market exclusivity. As an orphan medicine intended for pediatric use, LBS-008 may be eligible for additional incentives, such as an extension of its supplementary protection certificate for an additional two years of market exclusivity.
About Lin BioScience, Inc.
Lin BioScience, Inc. is a drug development company targeting untreatable diseases. Our therapeutic pipeline, which includes two candidates with Orphan Drug Status, is focused on meeting the unmet needs of patients suffering from cancer, macular degeneration, liver disease and diabetes.