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Amarantus Receives Orphan Drug Designation From the U.S. Food and Drug Administration for MANF for the Treatment of Retinal Artery Occlusion
Amarantus Receives Orphan Drug Designation From the U.S. Food and Drug Administration for MANF for the Treatment of Retinal Artery Occlusion.
About this update from Amarantus Bioscience Holdings, Inc.
[{"type":"text","content":"\nSAN FRANCISCO and GENEVA, Sept. 14, 2015 (GLOBE NEWSWIRE) -- Amarantus BioScience Holdings, Inc. (OTCQX:AMBS), a biotechnology company developing therapeutic and diagnostic product candidates in orphan indications and neurology, announced that the U.S. Food and Drug Administration (FDA) has granted the company's investigational drug mesencephalic-astrocyte-derived neurotrophic factor (MANF) orphan drug designation for the treatment of retinal artery occlusion (RAO). RAO is a blockage of the blood supply to the retina which causes severe and sudden loss of vision.\n\n\tMANF, a naturally-occurring protein that reduces and prevents apoptosis (programmed cell death) in response to injury or disease, was discovered utilizing Amarantus' proprietary PhenoGuard™ Protein Discovery Engine. Pre-clinical data demonstrated that MANF provided protective functional effects in an animal model of RAO. Moreover, toxicology studies have demonstrated that MANF was well-tolerated following a single intravitreal administration of a therapeutically relevant dose.\n\n\t\"We are extremely pleased to have received the second orphan drug designation for MANF for use in ophthalmologic disorders. At the end of 2014 we received orphan drug designation for MANF to treat retinitis pigmentosa (RP), and this additional designation for treatment of RAO is an important step in our regulatory strategy for what we believe has the potential to become a broad ophthalmic therapeutic franchise,\" said Gerald E. Commissiong, President & CEO of Amarantus BioSciences Holdings, Inc. \"RAO is an acute condition that can potentially lead to blindness and the toxicology data generated thus far directly supports the translational potential for MANF in this indication. We believe MANF has promise as a safe and effective therapeutic option to treat RAO and RP.\"\n\n\tThe FDA Orphan Drug Designation program provides a special status to drugs and biologics intended to treat, diagnose or prevent so-called orphan diseases and disorders that affect fewer than 200,000 people in the U.S. This designation provides for a seven-year marketing exclusivity period against competition, as well as certain incentives, including federal grants, tax credits and a waiver of PDUFA filing fees.\n\n\tAbout Retinal Artery Occlusion\n\n\tRetinal artery occlusion (RAO) is a rare eye condition...