The Food and Drug Administration (FDA) has four distinct approaches to expedite the availability of therapies for rare, severe, or life-threatening illnesses: Priority Review, Breakthrough Therapy, Fast Track, and Accelerated Approval. Fast Track designation is granted to drugs which can treat serious illnesses or conditions, or fill unmet needs for patients. Drugs which receive this status also gain benefits for the drug developers: more frequent meetings and written communication from the FDA, potential Priority Review and Accelerated Approval, and Rolling Review. According to a news release from early May 2022, LBS-008 (Tinlarebant), a therapy for Stargardt disease type 1, recently earned Fast Track designation. 

Learn more about Fast Track designation. 

LBS-008: An Overview

Developed by biopharmaceutical company Belite Bio, Inc. (“Belite”), LBS-008 is described as:

an oral once-a-day treatment that can reduce and maintain the delivery of vitamin A (retinol) to the eye as a means to reduce the accumulation of toxic vitamin A byproducts in ocular tissue, [preserving the integrity of retinal tissues and ultimately slowing or preventing vision loss].

LBS-008 is also being evaluated as a candidate for dry age-related macular degeneration. Outside of Fast Track designation, LBS-008 has received Orphan Drug and Rare Pediatric Disease designations in the United States, and Orphan Drug designation in Europe. 

Currently, Belite is evaluating LBS-008 in a Phase 3 clinical trial for Stargardt disease Type 1. Additionally, the company is planning to run a Phase 2/3 clinical trial to evaluate the therapy for dry age-related macular degeneration later this year. 

About Stargardt Disease

ABCA4 gene mutations are the most common cause of Stargardt disease, a rare inherited eye disease which affects the retina. Normally, this gene creates a protein that clears vitamin A byproducts out of photoreceptors. However, these gene mutations cause lipofuscin, a fatty yellow pigment, to build up inside of the macula. This causes cones and rods to die, resulting in progressive vision loss. Typically, vision loss associated with Stargardt disease begins during childhood or adolescence. However, some people may not notice vision loss until adulthood. Symptoms include:

• Progressive vision loss in both eyes
• Color blindness
• Loss of peripheral vision
• Involuntary eye movements
• Bright light sensitivity
• Delayed adjustment between light and dark environments
• Gray, black, or hazy spots in the center of vision