FDA investigate potential Stargardt treatment

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The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to a drug to help treat Stargardt disease, according to Lin BioScience.

The drug, known as LBS-008, is designed to prevent the build-up of a toxin in the eye called lipofuscin, that causes damage to the retina in Stargardt disease and dry age-related Macular Degeneration (AMD).

Stargardt disease is currently an untreatable inherited condition that causes permanent central vision loss during childhood and adolescence. It is the most common form of juvenile macular degeneration with an incidence of approximately 1 in 10,000 births.

Dr. Tom Lin, CEO of Lin Bioscience, said: “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.

"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.”

The drug is expected to enter first-in-human Phase 1 clinical trials this year for both dry AMD and Stargardt disease. For more updates from the FDA, including applications, designations and approvals, follow Rare Disease Report on Facebook and Twitter.

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.