QLT Inc. announced the company's oral synthetic retinoid for retinal diseases, QLT091001, has been granted two Fast Track designations by the US Food and Drug Administration (FDA) for the treatment of Leber Congenital Amaurosis (LCA) due to inherited mutations in LRAT and RPE65 genes and for the treatment of autosomal recessive Retinitis Pigmentosa (RP) due to inherited mutations in LRAT and RPE65 genes.
The FDA’s Fast Track is a process designed to facilitate the development and expedite the review of drugs that are intended for the treatment of serious diseases and fill an unmet medical need.
Following the recent completion of the QLT091001 phase I b trial in the LCA cohort, the Company continues the ongoing follow-up and initial re-treatment of LCA patients, as well as targeting the completion of subject enrollment in the RP cohort before the end of 2011.
QLT received clearance from the FDA in July 2011 of our Investigational New Drug Application (IND) for QLT091001 allowing the Company to proceed with investigator sites in the US for LCA and RP patients. Also in July, QLT received clearance from the UK Medicines and Healthcare Products Regulatory Agency of our Clinical Trial Application (CTA) for QLT091001 allowing the Company to proceed with investigator sites in that jurisdiction for LCA and RP patients.
“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 Bob Butchofsky, president and chief executive officer of QLT Inc. “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.”
The purpose of the FDA’s Fast Track process is to get important new drugs to the patient earlier. Once a drug receives Fast Track designation, early and frequent communication with the FDA is encouraged through the drug development and review process, potentially leading to earlier drug approval. In addition, a drug that receives Fast Track designation also qualifies for a Rolling Submission, which means that a company can submit completed sections of its New Drug Application (NDA) for review by the FDA, rather than waiting until the entire application is available before review can commence.
Genetic diseases in the eye such as LCA and RP arise from gene mutations of enzymes or proteins required in the biochemistry of vision. QLT091001 is a replacement for 11-cis-retinal, which is an essential component of the retinoid-rhodopsin cycle and visual function. QLT091001 has received orphan drug designations for the treatment of the LRAT and RPE65 genetic mutations in both LCA and RP by the US Food and Drug Administration. QLT091001 has also received orphan drug designations for the treatment of LCA and RP by the European Medicines Agency.
LCA is an inherited degenerative retinal disease characterized by abnormalities such as roving eye movements and sensitivity to light, and manifesting in severe vision loss from birth. Eye examinations of infants with LCA reveal normal appearing retinas. However, a low level of retinal activity, measured by electroretinography, indicates very little visual function. Approximately 1 child out of every 81,000 births will inherit the disease. Mutations in the genes for retinal pigment epithelium protein 65 (RPE65) and lecithin:retinol acyltransferase (LRAT) result in an inadequate production of 11-cis-retinal and occur in approximately 10% of patients with LCA and to a lesser extent in Retinitis Pigmentosa (RP), another inherited retinal dystrophy.
RP is a set of hereditary retinal diseases demonstrating clinical features similar to LCA and characterized by degeneration of rod and cone photoreceptors.
QLT is an ocular-focused company dedicated to the development and commercialization of innovative ocular products that address the unmet medical needs of patients and clinicians worldwide.