QLT (TSE:QLT) (NASDAQ:QLTI) announced Wednesday it received two fast track designations from the U.S. FDA for the speedy development of its QLT091001 drug, intended for the treatment of two degenerative eye diseases, Leber Congenital Amaurosis (LCA) and Retinitis Pigmentosa (RP).
Both LCA and RP are inherited genetic diseases, frequently caused by mutations to the LRAT and RPE65 genes. QTL's OTL091001 drug is an oral synthetic retinoid, and works by replacing the 11-cis-retinal chromophore, an essential part of visual function.
The FDA's fast track designation facilitates the quick development of investigational drugs that show promise in treating serious diseases, expediting the regulatory review process to fulfill a previously unmet medical need.
In May, QLT released the results of its phase 1b study of QLT091001 in the treatment of LCA. The results of the 12-patient clinical trial reported that eight subjects showed significant improvements in the study's endpoints - visual acuity and visual fields.
The company said it is continuing to follow up with the 12 patients from its phase 1b study, and intends to complete subject enrollment for the RP-based study by the end of the year.
"QLT091001's Fast Track designation, along with its orphan drug designation, highlights the need for therapeutic options for those patients suffering from inherited retinal diseases," said president and CEO, Bob Butchofsky.
"We are pleased that the FDA recognizes QLT091001 as a potential treatment for LCA and RP and we are now working diligently to advance the development of this compound under the Fast Track process."
Characterized by abnormalities like roving eye movements and sensitivity to light, and manifested in severe vision loss beginning at birth, LCA affects an estimated one infant out of every 81,000 births.
Meanwhile, RP is characterized by the degeneration of photoreceptors in the eye, and affects at least 300,000 people across the globe.
Based in Vancouver, B.C., ocular-focused medical company QLT's stock closed on Tuesday at $7.20 per share.