NEW YORK, NY--(Marketwired - May 20, 2015) - PlasmaTech Biopharmaceuticals, Inc. (PTBI), a biopharmaceutical company focused on gene therapy and plasma-based products for severe and life- threatening rare diseases announced today that the U.S. Food and Drug Administration (FDA) had granted both Orphan Drug Designation and Rare Pediatric Disease Designation for both of PlasmaTech Biopharmaceuticals' lead product candidates for the treatment of Sanfilippo Syndromes A and B.

"We are very pleased to announce that the FDA has provided these two important designations to our Sanfilippo drug candidates, reflecting their potential to address the significant unmet medical needs of children with these rare and devastating lysosomal storage diseases," stated Tim Miller, Ph.D., President & CEO of PlasmaTech Biopharmaceuticals. "The benefits and incentives associated with these designations, including marketing exclusivity periods and the potential to obtain two valuable Pediatric Disease Priority Review vouchers, are strategically important from a regulatory and commercial perspective and potentially very value-creating for shareholders."

"These important FDA designations reflect the critical nature of these two programs to these patients and families," noted Steven H. Rouhandeh, PlasmaTech's Executive Chairman. "PlasmaTech's programs in MPS IIIB & IIIA address compelling unmet medical needs with breakthrough technology. We are committed to building on these achievements, and intend to continue to expand our product pipeline in the rare disease space."

About Orphan Drug Designation: Under the FDA's Orphan Drug Designation program, orphan drug designation is granted by the FDA to novel drugs or biologics that treat rare diseases or conditions affecting fewer than 200,000 patients in the U.S. The designation allows the drug developer to be eligible for a seven-year period of U.S. marketing exclusivity upon approval of the drug, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical trial design assistance, and the waiver of Prescription Drug User Fee Act (PDUFA) filing fees.

About the Pediatric Disease Priority Review Voucher Program: 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.

About FDA Standard Review and Priority Review Designations

Prior to approval, each drug marketed in the United States must go through a detailed FDA review process. The FDA agreed, under PDUFA in 1992, to specific goals for improving the review time of NDAs and BLAs and created a two-tiered system of review times -- Standard Review and Priority Review. Standard Review can be accomplished in a ten-month time frame from the time the application is filed by the FDA, which typically occurs approximately 60 days following submission of the application. A Priority Review designation is given to drugs that offer major advances in treatment, or provide a treatment where no adequate therapy exists. The FDA goal for reviewing a drug with Priority Review status is six months from the time the application is filed by the FDA.

About Sanfilippo Syndrome: Sanfilippo Syndrome is a group of 4 deadly genetic diseases resulting from the body's inability to properly break down certain sugars. In the class of lysosomal storage diseases, symptoms often appear in the first year of life, and the disease causes progressive muscular and cognitive decline in children after the age of two. Children afflicted with Sanfilippo Syndrome experience progressive loss of speech, the ability to eat and walk, and rarely live past their second decade of life. There is no cure and currently no approved treatments for Sanfilippo syndrome.

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