Advertisement PlasmaTech's lead product candidates get FDA orphan drug and rare pediatric disease designations - Pharmaceutical Business review
Pharmaceutical Business review is using cookies

ContinueLearn More
Close

PlasmaTech’s lead product candidates get FDA orphan drug and rare pediatric disease designations

PlasmaTech Biopharmaceuticals (PTBI), a biopharmaceutical company focused on gene therapy and plasma-based products for severe and life- threatening rare diseases announced that the US 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."

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 US.

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.