Live Biotherapeutic Directed at Rare Disease Affecting Very Low Birthweight Babies
GAITHERSBURG, MD, April 1, 2015 – Sigma-Tau Pharmaceuticals, Inc., announced today that the U.S. Food and Drug Administration (FDA) has granted an orphan drug designation for the Company’s investigational compound, STP-206 (lactobacillus acidophilus and Bifidobacterium animalis subsp. lactis), a live biotherapeutic being developed for the prevention of necrotizing enterocolitis (NEC) in premature infants with very low birth weight less than or equal to 1,500 grams. The Company is currently conducting a multi-center, double-blind, randomized, placebo-controlled Phase 1b/2a clinical trial for STP-206 at a number of different sites in the United States.
“Today’s announcement marks the second orphan drug designation received by our Company in the last week, and as a leader in the pioneering field of live biotherapeutics, we continue to work diligently to bring additional, innovative therapies to market,” commented Gianfranco Fornasini, Senior Vice President of Scientific Affairs of Sigma-Tau Pharmaceuticals, Inc.
"NEC is a very serious disease for which there is currently no approved treatment,” said Dave Lemus, Chief Executive Officer of Sigma-Tau Pharmaceuticals, Inc. "Moreover, we are encouraged by the achievement of this important regulatory milestone, and remain resolute in our commitment to address the unmet medical needs of those suffering from rare diseases.”
An Orphan drug designation is granted by the FDA Office of Orphan Products Development to novel drugs that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides the beneficiary company with a seven-year period of U.S. marketing exclusivity if it is the first product to reach marketing approval, as well as potential tax credits for clinical research costs, federal grants, and a waiver of Prescription Drug User Fee Act (PDUFA) filing fees.
Necrotizing enterocolitis (NEC) is a rare gastrointestinal disease affecting premature babies mainly with very low birth weight (infants weighing less than or equal to 1,500 grams or 3.3 pounds). The condition involves inflammation of the bowel that can lead to perforation of the intestine, severe infection, shock, and death. NEC is reported in approximately 10% of very low birth weight babies or approximately 4,200 occurrences per year in the U.S. The reported death rate from NEC is in the range of 20-30%, or approximately 1,000 deaths a year in the U.S. There is presently no FDA-approved treatment or prevention for NEC, resulting in an urgent, unmet medical need.
About Live Biotherapeutics
A live biotherapeutic is a biological product that: (1) contains live microorganisms, such as bacteria or yeast, that are naturally occurring, recombinant, or clonally selected; (2) is applicable to the prevention, treatment, or cure of a disease or condition of human beings; and (3) is not an immunogen-specific vaccine. As the characterization of the human microbiome and its link to human health has become better understood, the use of live biotherapeutic products in clinical application has shown promise for reducing infection, stimulating innate immune responses, and modulating gastrointestinal metabolism.
About Sigma-Tau Pharmaceuticals, Inc.
Sigma-Tau Pharmaceuticals, Inc. is a U.S. based, wholly owned subsidiary of the sigma-tau Group, and is dedicated to the global development and commercialization of medicines for patients with rare diseases. Sigma-Tau Pharmaceuticals, Inc. is based in Gaithersburg, Maryland, and the company’s marketed products are focused on cancer, kidney disease, gastrointestinal and genetic related disorders. The company also has clinical development programs focused on hematological cancer, malaria, and other areas of unmet medical need. For more information about Sigma-Tau Pharmaceuticals, Inc., visit www.sigmatau.com.
Contact at Sigma-Tau:
Dave Lemus (CEO)
Contact for Sigma-Tau (Media):
Rx Communications Group, LLC