PlasmaTech Biopharmaceuticals Announces Orphan Drug and Rare Pediatric Disease Designations from FDA Print
Wednesday, 20 May 2015 14:03

NEW YORK, NY--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.

About PlasmaTech Biopharmaceuticals: PlasmaTech Biopharmaceuticals is focused on advancing gene therapy and plasma-based products for rare diseases. PlasmaTech's lead program is a gene therapy for Sanfilippo syndrome (MPS IIIA and IIIB) in collaboration with patient advocate groups, researchers and clinicians. Clinical trials for Sanfilippo types A and B are anticipated to begin in 2015.

In addition, the company is pursuing two additional proprietary platforms, Salt Diafiltration (SDF™) Process and Polymer Hydrogel Technology (PHT™), and is active in the development and commercialization of human plasma-derived therapeutics, including its proprietary alpha-1 protease inhibitor, SDF Alpha™. The company has developed a robust product pipeline that includes two commercial stage products, MuGard® and ProctiGard™, with additional follow-on products in development. For more information, visit

This press release contains certain statements that are forward-looking within the meaning of Section 27a of the Securities Act of 1933, as amended, and that involve risks and uncertainties. These statements include, without limitation, those relating to: the Company's acquisition of Abeona, anticipated acceleration in the development and internationalization of clinical programs, information regarding the future performance of the combined company, the outlook on medical needs, future pipeline expectations, management plans for the Company, the anticipated closing of the transaction, and general business outlook. These statements are subject to numerous risks and uncertainties, including but not limited the satisfaction of closing conditions for the transaction, the parties' ability to successfully integrate and operate the new company, and achieve expected synergies and other benefits; the impact of competition; the ability to develop products and technologies; the ability to achieve or obtain necessary regulatory approvals; the impact of changes in the financial markets and global economic conditions; and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and other reports filed by the Company with the Securities and Exchange Commission. The Company undertakes no obligations to make any revisions to the forward- looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

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