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Sanfilippo Syndrome Gene Therapy: PlasmaTech Biopharmaceuticals’ Rare Pediatric Disease Vouchers

May 20, 2015

PlasmaTech Biopharmaceuticals, a Dallas-based biopharmaceutical company focusing on protein biologic therapies and oncology supportive care products, announces on May 20th, that the FDA has granted Rare Pediatric Disease Designations to both of their investigational Gene Therapy products for Sanfilippo Syndrome (SF), MPS IIIB and MPS IIIA:

•   Gene Therapy product ABX-101 for MPS IIIB

•   Gene Therapy product ABX-102 for MPS IIIA.

Per the news release:

“Under the FDA’s Pediatric Disease Priority Review Voucher program, upon the approval of a qualifying new drug application (NDA) or biologics license application (BLA) for the treatment of a rare pediatric disease, the sponsor of such application would be eligible for a Pediatric Disease Priority Review Voucher that can be used to obtain priority review for a subsequent NDA or BLA. The 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. The Priority Review Voucher may be sold or transferred an unlimited number of times.”

PlasmaTech Biopharmaceuticals obtained both gene therapy products as a result of the company’s recent completion of the acquisition of Abeona Therapeutics. In April 2014, the FDA grants Abeona Therapeutics’ two gene therapies for Sanfilippo Syndrome Types A and B, Orphan Drug Designation (ODD):

 # Generic Name/ODD Date Sponsor Company Indication
1 Recombinant AAV9 expressing human sulfoglucosamine sulfohydrolase/ 04.29.14 Abeona Therapeutics MucopolysaccharidosisType III-A(Sanfilippo SyndromeType A)
2 Recombinant AAV9 expressing human alpha-N-acetylglucosaminidase/ 04.30.14 Abeona Therapeutics MucopolysaccharidosisIII-B(Sanfilippo SyndromeType B)


Please Note: “DNA Repair” courtesy of Tom Ellenberger, Washington University School of Medicine in St. Louis. [Public domain] | Wikimedia Commons

Copyright © 2012-2015, Orphan Druganaut Blog. All rights reserved.

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