Synspira Therapeutics’ investigational inhaled treatment, PAAG15A, has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of cystic fibrosis.

PAAG15A, also known as SNSP113, is under development by Synspira Therapeutics as an innovative inhaled therapy designed to enhance lung function in patients with cystic fibrosis. This drug targets the primary contributors to pulmonary deterioration—namely, infection, airway congestion, and inflammation.

Cystic fibrosis is a progressive genetic disorder characterized by the accumulation of thick, sticky mucus in the lungs and airways. This condition causes airway obstruction, prolonged lung infections, and chronic inflammation, leading to significant lung damage and potentially respiratory failure.

FDA Orphan Drug Designation Highlights Need for New Treatment Options

Synspira Therapeutics’ PAAG15A has now received orphan drug designation from the FDA, a status granted to drugs intended to treat rare diseases. Cystic fibrosis affects over 30,000 people in the United States and an equal number in Europe, according to the Cystic Fibrosis Foundation.

The disease’s hallmark is the buildup of thick mucus that obstructs airflow and fosters bacterial infections. These infections are often exacerbated by bacteria forming protective biofilms, which are resistant to antibiotic penetration and can lead to the growth of multi-drug-resistant strains.

SNSP113: Targeting the Root Causes of Pulmonary Decline

Synspira’s SNSP113 aims to address these challenges by specifically targeting and disrupting bacterial biofilms. By doing so, it enhances the effectiveness of antibiotics and helps in normalizing the thick mucus. This mechanism could potentially offer relief to a broad population of cystic fibrosis patients, as it treats the factors driving pulmonary decline rather than the underlying genetic mutation associated with cystic fibrosis.

Shenda Baker, CEO of Synspira Therapeutics, commented on the significance of the FDA’s orphan designation, stating, “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.”

Baker also highlighted the broad applicability of SNSP113, noting, “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 up to $3 million from the Cystic Fibrosis Foundation to advance the clinical development of SNSP113, emphasizing the drug’s potential in addressing pulmonary complications associated with cystic fibrosis.

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