FRAMINGHAM, Mass., April 10, 2019 —Synspira Therapeutics today announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has granted Orphan Drug Designation to the Company’s lead product, SNSP113, a first-in-class glycopolymer, for the treatment of people with cystic fibrosis (CF). SNSP113 was granted Orphan Designation for the treatment of CF by the U.S. Food and Drug Administration (FDA) in October 2018. SNSP113 targets the key drivers of pulmonary decline in cystic fibrosis and other progressive pulmonary diseases, including life-threatening pulmonary infections.
The company’s lead product, SNSP113, is a first-in-class inhaled glycochemistry-based therapeutic with a broad spectrum mechanism of action intended to target the underlying cascade of events that lead to progressive pulmonary disease or other life-threatening pulmonary conditions, such as pulmonary infections with nontuberculous Mycobacteria (NTM), Burkholderia cepacia complex (BCC), Pseudomonas aeruginosaor methicillin-resistant Staphylococcus aureus(MRSA). SNSP113 is designed to normalize mucin viscosity and improve mucus transport to increase airway clearance. SNSP113 disrupts the cohesion of bacterial biofilms and interacts with the cell walls of invading bacteria to increase their permeability, reduce their viability and potentiate the efficacy of antibiotics. These actions of SNSP113 lead to a reduction in the inflammatory cascade of neutrophils that can lead to pulmonary damage and fibrosis. Progressive pulmonary disease leads to overwhelming symptoms, impacts quality of life (QoL) and results in debilitating progressive lung decline.
Synspira Therapeutics is a clinical-stage biopharmaceutical company dedicated to significantly improving the lives of people with cystic fibrosis and other rare diseases, such as primary ciliary dyskinesia and bronchiectasis, where there is a high unmet treatment need. Synspira is a privately held company headquartered in Framingham, MA. The company’s lead product, SNSP113, is a first-in-class inhaled glycochemistry-based therapeutic with a broad-spectrum mechanism of action designed to target the underlying cascade of events that lead to progressive pulmonary disease or other life-threatening pulmonary conditions. At Synspira we are inspired by the patients we serve and are driven to make a difference.