Grifols, a global healthcare company based in Barcelona, Spain, plans to initiate a safety trial of a novel, inhaled formulation of alpha1-proteinase inhibitor [human] later this year.  In April, the FDA granted orphan drug designation for Grifols' inhaled alpha1 formulation as a treatment for cystic fibrosis, an inherited disease that can cause life-threatening lung infections. Orphan drug designation is granted to encourage the development of treatments that prevent, diagnose or treat rare diseases that affect fewer than 200,000 people per year in the U.S.

"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. "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 currently has the intravenous therapy PROLASTIN-C (Alpha1-Proteinase Inhibitor [Human])(A1-PI), indicated for the treatment of alpha1-antitrypsin (AAT) deficiency. This rare, genetic disorder can result in the development of emphysema due to low circulating levels of the alpha1 protein in the lungs.

PROLASTIN, the predecessor to PROLASTIN-C, was the first FDA-approved product to treat AAT deficiency and remained so for nearly 17 years. Grifols is developing its next-generation alpha­1-proteinase inhibitor as an inhaled formulation.