Synspira has been given the orphan designation status by the US Food and Drug Administration (FDA) for poly (acetyl, arginyl) glucosamine (PAAG15A) for cystic fibrosis treatment.
PAAG15A is being developed by Synspira as as SNSP113, an investigational inhaled treatment to boost lung function in cystic fibrosis patients by targeting the key drivers of pulmonary decline, which are infection, airway congestion and inflammation.
Considered to be a progressive genetic disease, cystic fibrosis creates airway obstruction, prolonged lung infections and chronic inflammation of pulmonary tissue. The genetic disease can result in permanent lung damage and eventually cause respiratory failure.
Cystic fibrosis is distinguished by the build-up of thick, sticky mucus in the lungs and clogged airways which hamper breathing.
Bacteria are not cleared easily and create protective biofilms that are not easy for antibiotics to penetrate and often results to the growth of multi-drug resistant bacteria. Over 30,000 people in the US, and an identical number in Europe, suffer from cystic fibrosis according to the Cystic Fibrosis Foundation.
Synspira, which is focused on developing inhaled glycopolymer-based therapeutics for pulmonary disease treatment, says that the investigational cystic fibrosis treatment SNSP113 can specifically target and disrupt the cohesion of bacterial biofilms. Thereby, it increases the chances of the activity of antibiotics and normalizes thick mucus.
Commenting on the FDA orphan designation for the investigational cystic fibrosis treatment PAAG15A, Shenda Baker – CEO of Synspira, said: “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.
Shenda Baker further said: “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.”
Earlier this year, Synspira secured an award up to $3 million from the Cystic Fibrosis Foundation to advance clinical development of SNSP113 for the treatment of pulmonary complications of cystic fibrosis.