Grifols, Barcelona, Spain, announced plans to initiate a safety trial of a novel, inhaled formulation of alpha1-proteinase inhibitor (Human) later this year. In April, the US Food and Drug Administration (FDA) granted orphan drug designation for Grifols' inhaled alpha1 formulation as a treatment for cystic fibrosis.
"Grifols is committed to developing new therapies that address the debilitating symptoms of chronic lung disease," said Kim Hanna, vice president of clinical research development at Grifols. "The orphan drug designation represents another milestone in the growth of our alpha1 program, and we're excited to pursue clinical trials with an aerosol formulation of this important therapy."
Grifols also produces the alpha1-proteinase inhibitor PROLASTIN®-C (Alpha1-Proteinase Inhibitor [Human])(A1-PI), an intravenous therapy indicated for the treatment of alpha1-antitrypsin (AAT) deficiency.
Source: Grifols .
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A1PI in Cystic Fibrosis: US Orphan Drug Designation
• CF is the most common fatal hereditary disorder that affects
individuals of European descent.
• Preclinical evidence indicates that proteinase activity may
play a central role in the pathophysiology of CF.
• A1PI may have the potential to slow the decline in lung
function and improve the quality of life in patients by reducing
inflammatory activity in the lung.