Synspira’s drug receives FDA Orphan Designation for Cystic Fibrosis treatment

ddd1401-orphan-drugs

Synspira,a privately held company developing a new class of inhaled glycopolymer-based therapeutics for the treatment of pulmonary disease, announced that it has been granted Orphan Designation by the United States Food and Drug Administration (FDA) for poly (acetyl, arginyl) glucosamine (PAAG15A), for the treatment of cystic fibrosis. Synspira is developing PAAG15A as SNSP113, a potential inhaled treatment to improve lung function in patients with cystic fibrosis (CF).

“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 Burkholderiaand 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.”

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 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 U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.

 

About Pharmascroll:

Pharmascroll is a diligent business consulting and market research firm focused solely towards pharmaceutical markets. The company consults and researches in majorly chronic disease indications prevalent across the globe. The research conducted by Pharmascroll analysts is targeted to provide analytical and logical answers to the key business questions of the pharmaceutical and medical insights teams and to make better informed business decisions with the detailed relevant information available.

To read more news about Cystic Fibrosis, visit https://pharmascroll.com/news-category/cystic-fibrosis/

 

News Source: Synspira website

Image Source: https://www.rdmag.com/article/2014/01/orphans-no-longer