Synlogic Proprietary Synthetic Biotic Receives FDA Orphan Drug Designation
Tuesday, August 30, 2016
Synlogic, a privately-held biopharmaceutical company developing novel medicines based on its proprietary synthetic biology and microbiome platform, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Synlogic's investigational program involving a live E. coli Nissle bacterium modified to assimilate ammonia for treatment of urea cycle disorders (UCDs). UCDs are a family of rare but serious genetic mutations that allow the build-up of toxic levels of ammonia in the bloodstream, causing severe neurological complications and even death. Synlogic’s lead candidate, SYNB1020, is currently in preclinical development and the company plans to file an investigational new drug application (IND) with the FDA for the potential treatment of UCDs in the first quarter of 2017.
“We are pleased that the FDA recognizes the significant need that exists among children and adults living with UCD,” said Jose Carlos Gutierrez-Ramos, CEO of Synlogic. “Receiving orphan drug designation is an important step forward in our shared goal of bringing this novel treatment approach to the patients and families affected by urea cycle disorders. We look forward to evaluating our lead candidate SYNB1020 for the potential treatment of this debilitating disease.”
The FDA’s Orphan Drug program offers orphan status to drugs and biologics that are intended for the treatment of rare diseases affecting fewer than 200,000 people in the U.S. The designation provides sponsors with development and commercial incentives for designated compounds and medicines, including eligibility for a seven-year period of market exclusivity in the U.S. after product approval, FDA assistance in clinical trial design and an exemption from FDA user fees.
Source : businesswire.com
