New drug for Stargardt could reach patients fasterPosted: Friday 17 June 2022 at 08:39
A new drug to treat Stargardt disease has been granted a fast-track review by the Food and Drug Administration in the US, to help make it available to patients quicker.
The drug, known as LBS-008, is an orally administered tablet and has been trialled in patients over a 12-month period, as part of a phase two clinical study by US-based pharmaceutical company, Belite Bio.
It is hoped the tablet, which is now in phase three trials, will halt or slow the progression of Stargardt, one of the most common forms of inherited macular disease, which there is currently no treatment for.
Belite Bio said the new drug prevents the build-up of toxins in the eye that cause Stargardt disease. These toxins are by-products of the visual cycle, a series of chemical reactions which happen when light hits the retina. The visual cycle is dependent on the supply of vitamin A (retinol) to the eye. LBS-008 works by reducing and maintaining levels of a particular protein which is responsible for transporting retinol to the eye. By controlling the amount of retinol entering the eye, LBS-008 reduces the formation of toxins in order to maintain the health of retinal tissues.
The drug is also being trialled for use in patients with dry age-related macular degeneration, with the company planning to launch a Phase two/three clinical trial later this year.
Speaking about the FDA’s decision to grant the drug Fast Track Designation, Dr. Tom Lin, Belite Bio’s Chairman and CEO said: “We are delighted that LBS-008 has received FDA Fast Track Designation.
“STGD1 is a terrible retinal disease with the potential to severely affect the vision of afflicted patients and there are currently no approved treatments.
“At present, we are conducting a Phase three clinical trial in order to bring to market a treatment that will halt or slow the progression.”
The results from the phase 2 trial are expected at the end of 2022 when all patients have completed 12 months of treatment.
Moorfields Eye Hospital is taking part in the phase three trials for LBS-008. Register to find out more about
Research Opportunities at Moorfields (ROAM) and be contacted about research studies relevant to you.
What does Fast Track Designation mean?
Fast Track Designation is a process designed to facilitate the development, and accelerate the review, of drugs which show promise to treat serious conditions and fill an unmet medical need.
The request for a Fast Track Designation can be made by the drug company at any time during the drug development process. But the drug will still need to go through the usual phases of trials and be shown to be safe and effective.
The purpose is to get important new drugs to the patient earlier.
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