A cystic fibrosis therapeutic PAAG15A has been granted Orphan Designation by the US FDA, according to manufacturer Synspira. PAAG15A, also known as SNSP113.
SNSP113 is the lead candidate being developed by Synspira as part of a new class of proprietary glycopolymer-based therapeutics. SNSP113 is designed to specifically target and disrupt the cohesion of bacterial biofilms, potentiating the activity of antibiotics, and normalizing thick mucus. SNSP113 is intended to improve lung function in CF patients by targeting the key drivers of pulmonary decline – infection, airway congestion and inflammation.
“The orphan designation granted for PAAG15A by the FDA reflects the need for new treatment options for patients living with cystic fibrosis, and in particular for those patients with relentless and antibiotic resistant bacterial infections such as Burkholderia and non-tuberculous Mycobacteria,” said Shenda Baker, Ph.D., Chief Executive Officer of Synspira. “We are developing SNSP113 to treat pulmonary infection and airway congestion in cystic fibrosis patients to improve pulmonary health. Because SNSP113 treats the drivers of pulmonary decline rather than the underlying CFTR mutation, it has the potential to treat a broad population of CF patients regardless of genetic mutation.”