Therapy Areas: Hereditary Disorders
Synspira Receives US FDA Orphan Designation for PAAG15A for the Treatment of Cystic Fibrosis
11 October 2018 - - US-based inhaled glycopolymer-based therapeutics developer Synspira has been granted Orphan Designation by the United States Food and Drug Administration for poly (acetyl, arginyl) glucosamine (PAAG15A), for the treatment of cystic fibrosis, the company said.

Synspira is developing PAAG15A as SNSP113, a potential inhaled treatment to improve lung function in patients with cystic fibrosis.

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 normalising 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 Drug Designation Program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the US, or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.

SNSP113 is a glycopolymer-based therapeutic being developed as an inhaled treatment to improve lung function in patients with cystic fibrosis.

PAAG15A is the active modified polysaccharide in SNSP113 that interacts with structural polymers in protective bacterial biofilms, breaking them apart, and with native glycoproteins in mucus, normalising mucus viscosity.

SNSP113 also interacts with the cell walls of invading bacteria increasing their permeability, thereby reducing their inherent viability and potentiating the efficacy of antibiotics. SNSP113 is designed to reduce infection, airway congestion and inflammation, the key drivers of pulmonary exacerbations and pulmonary decline in cystic fibrosis patients.

Cystic fibrosis is a progressive genetic disease that causes airway obstruction, persistent lung infections and chronic inflammation of pulmonary tissue leading to permanent lung damage and ultimately resulting in respiratory failure.

CF is characterised by the accumulation of thick, sticky mucus in the lungs and clogged airways which impede breathing.

Bacteria are not easily cleared and create protective biofilms that are difficult for antibiotics to penetrate and often lead to the emergence of multi-drug resistant bacteria.

More than 30,000 people in the United States, and a similar number in Europe, live with cystic fibrosis.

Synspira is developing a new class of inhaled glycopolymer-based therapeutics to reduce pulmonary inflammation, airway obstruction and infection, key drivers of pulmonary diseases including cystic fibrosis, chronic obstructive pulmonary disease, and pneumonia.

The company has an exclusive license from Synedgen to the Glycomics Technology Platform for the development of inhaled therapeutics in pulmonary indications. Synspira is dedicated to developing drugs with new mechanisms of action to target and change the course of pulmonary diseases.


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