Amarantus Applies to FDA for MANF Orphan Drug Designation in Retinitis Pigmentosa
SAN FRANCISCO and GENEVA, Oct. 16, 2014 (GLOBE NEWSWIRE) -- Amarantus Bioscience Holdings, Inc. (OTCQB:AMBS), a biotechnology company focused on the development of diagnostics and therapeutics for Alzheimer's disease, Parkinson's disease and ophthalmological disorders, announced that it has applied to the US Food & Drug Administration for Orphan Drug Designation for MANF (mesencephalic-astrocyte-derived neurotrophic factor) in retinitis pigmentosa (RP). MANF was discovered utilizing Amarantus' proprietary PhenoGuard Protein Discovery Engine; all intellectual property is owned by the company.
MANF, a naturally-occurring protein that reduces and prevents apoptosis (programmed cell death) in response to injury or disease, has demonstrated efficacy in three animal models of retinitis pigmentosa. Amarantus is collaborating with the University of Miami's Bascom Palmer Eye Institute and the Buck Institute for Research on Aging in its retinitis pigmentosa program. Additionally, the company has completed toxicology experiments, concluding that MANF was well-tolerated following intravitreal administration at therapeutically relevant doses.
About Retinitis Pigmentosa
Retinitis pigmentosa (RP) refers to a group of inherited diseases causing retinal degeneration often leading to blindness. The cell-rich retina lines the inside back wall of the eye and is responsible for capturing images from the visual field. People with RP experience a gradual decline in their vision because photoreceptor cells (rods and cones) die. Symptoms include a progressive degeneration of peripheral and night vision, as well as the degeneration in color perception and central vision. Night blindness is one of the earliest and most frequent symptoms of RP.
RP is typically diagnosed in adolescents and young adults. The rate of progression and degree of visual loss varies from person to person. Most people with RP are legally blind by age 40. It is estimated that the market opportunity for Retinitis Pigmentosa exceeds $10B annually.
