FDA Orphan Drug Designation Granted to Potential Stargardt Treatment

Mathew Shanley

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to LBS-008 for the treatment of Stargardt Disease, Lin BioScience announced this morning.

LBS-008, a first-in-class oral therapy is designed to prevent the buildup of toxins in the eye that cause Stargardt Disease and atrophic Age-related Macular Degeneration (dry AMD).

Stargardt disease – also known as fundus flavimaculatus or juvenile macular degeneration – is a genetic condition that leads to progressive vision loss, mostly in children. The majority of patients become visually impaired by age 20. The most common form of the disease is the result of a mutation in the ABCA4 gene that causes an accumulation of toxic vitamin A byproducts in the retina which gradually destroy photoreceptors.

Per the National Institute of Health (NIH), approximately 1 in 8-10,000 people have Stargardt disease. At present, no therapies have been approved by the FDA to treat the condition.

“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 in a press release. “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 reduces circulating levels of retinol-binding protein (RBP4) and retinol (vitamin A1) in the body, which in turn  decreases uptake of retinol into the eye and the unwanted accumulation of its toxic byproducts.

The NIH’s Blueprint Neurotherapeutics Network has funded both discovery and development for the therapy, and will continue to provide financial support through the completion of Phase 1 clinical trials.

LBS-008 is expected to enter first-in-human Phase 1 clinical trials in 2017 for both dry AMD and Stargardt disease.

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