The United States Food and Drug Administration has awarded Rare Paediatric Disease Designation to an experimental drug for the treatment of Stargardt disease. For more information, you can find the original press release here, at PR Newswire.
About Stargardt Disease
Stargardt disease is a heritable disorder that can cause vision loss. The condition typically affects the retina (a part of the eye that senses light), leading to vision loss that often occurs in childhood or adolescence, although in some cases it may only become noticeable in adulthood.
One common cause of Stargardt disease is mutations in the gene ABCA4. This gene is involved in the production of a protein that helps to maintain healthy eyes. However, in some people with Stargardt disease, changes to this gene mean that the protein doesn’t work properly and as a result, a fatty substance can build up in the retina, affecting a person’s vision.
The Potential Treatment
An investigational drug is being developed as a potential treatment for Stargardt disease. The drug, called LBS-008, is thought to prevent toxins from building up in the eye (causing Stargardt disease). The NIH’s Blueprint Neurotherapeutics Network funded the discovery and development of this therapy.
Both the US and EU have awarded LBS-008 Orphan Drug status.
Rare Pediatric Disease Designation
Lin BioScience has recently announced that LBS-008 has also been awarded Rare Pediatric Disease Designation in the US. This designation, which is reserved for serious rare conditions that affect people aged eighteen or younger, may qualify Lin BioScience for a Priority Review Voucher if LBS-008 is approved by the FDA.
For more information, the source press release can be viewed here.