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Amarantus Requests Rare Pediatric Disease and Orphan Drug Designations from US FDA for Engineered Skin Substitute in the Treatment of Giant Congenital Melanocytic Nevi
Amarantus Requests Rare Pediatric Disease and Orphan Drug Designations from US FDA for Engineered Skin Substitute in the Treatment of Giant Congenital Melanocytic Nevi.
About this update from Amarantus Bioscience Holdings, Inc.
[{"type":"text","content":"\n\n\n\nAmarantus Requests Rare Pediatric Disease and Orphan Drug Designations from US FDA for Engineered Skin Substitute in the Treatment of Giant Congenital Melanocytic Nevi\n\n\n\n\n\nAmarantus Requests Rare Pediatric Disease and Orphan Drug Designations from US FDA for Engineered Skin Substitute in the Treatment of Giant Congenital Melanocytic Nevi\nPR Newswire\nSAN FRANCISCO, December 29, 2015\n\n\n\nSAN FRANCISCO, December 29, 2015 /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) and Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) to treat GCMN with Engineered Skin Substitute (ESS). It is estimated that the incidence of GCMN, a rare dermatological condition present at birth, is between 8 and 80 births annually in the United States.\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\nThe FDA Orphan Drug Designation program provides a special status to drugs and biologics intended to treat, diagnose or prevent 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\nAbout Giant Congenital Melanocytic Nevus \n\nGiant Congenital Melanocytic Nevus, a rare pediatric condition (also known as \"Bathing trunk nevus,\" \"Garment nevus,\" \"Giant hairy nevus\", and \"Nevus pigmentosus et pilosus\"), is defined by one or more large, darkly pigmented and sometimes hairy patches. The congenital (present a...