September 14, 2011 - The FDA has granted the biopharmaceutical company QLT with Fast Track Designations for its synthetic retinoid treatment for people with Leber congenital amaurosis (LCA) and retinitis pigmentosa (RP) caused by variations in the RPE65 and LRAT genes. Individuals with these genetic variations do not produce a retinoid critical for vision; QLT’s treatment serves as a replacement for that missing retinoid.
The Fast Track Designations can facilitate development and expedite the regulatory review of the treatment, potentially enabling it to reach patients sooner. This designation is given to drugs and therapies that address a serious, unmet medical need.
In spring 2011, QLT announced impressive one-year results for their 12-participant Phase IB clinical trial of their emerging synthetic retinoid therapy for people with LCA. All patients responded well to the seven-day treatment, reporting some gains in acuity and/or size of their visual field. The investigators were encouraged that the effect of the week-long oral treatment persisted for several months.
The company plans to enroll a total of 14 RP and 14 LCA patients in the trial by the end of 2011. |
