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Amarantus Requests Rare Pediatric Disease Designation from US FDA for MANF in the Treatment of Retinitis Pigmentosa
Amarantus Requests Rare Pediatric Disease Designation from US FDA for MANF in the Treatment of Retinitis Pigmentosa.
About this update from Amarantus Bioscience Holdings, Inc.
[{"type":"text","content":"\n\n\n\nAmarantus Requests Rare Pediatric Disease Designation from US FDA for MANF in the Treatment of Retinitis Pigmentosa\n\n\n\n\n\nAmarantus Requests Rare Pediatric Disease Designation from US FDA for MANF in the Treatment of Retinitis Pigmentosa\nPR Newswire\nSAN FRANCISCO, February 5, 2016\n\n\n\nSAN FRANCISCO, February 5, 2016 /PRNewswire/ --\n\nAmarantus BioScience Holdings, Inc. (OTCQB: AMBS), a biotechnology company focused on developing products for Regenerative Medicine, Neurology and Orphan Diseases, announced that it has requested Rare Pediatric Disease Designation (RPDD) from the US Food and Drug Administration (FDA) for treating retinitis pigmentosa (RP) with MANF. MANF was previously granted orphan drug designation (ODD) by the US FDA in December 2014.\n\nThe FDA defines a \"rare pediatric disease\" as a disease that affects fewer than 200,000 individuals in the U.S. primarily aged from birth to 18 years. Under the FDA's Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives an approval of a new drug application (NDA) or biologics license application (BLA) for a rare pediatric disease may be eligible for a voucher, which can be redeemed to obtain expedited FDA review for any subsequent marketing application. Vouchers  may be sold or transferred by the recipient; in the last 6 months, 2 priority review vouchers have been sold for a combined $595M in cash.\n\nIn December 2015, Amarantus submitted ODD and RPDD applications to the US FDA for engineered skin substitute in the treatment of Giant Congenital Hairy Nevus (GCMN), in addition to its ODD application to the US FDA for eltoprazine in the treatment of Parkinson's disease levodopa-induced dyskinesia (PD-LID) submitted in October 2015. The Company expects to receive responses regarding these designation applications in the first quarter of 2016.\n\nAbout Retinitis Pigmentosa \n\nRetinitis pigmentosa (RP) refers to a group of inherited diseases causing retinal degeneration often leading to blindness. The retina lines the back inside 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 the retinal cells responsible for black-white and color perception die in response to stress. As a result, the telltale presenting symptoms often include n...