(By Balaseshan) Grifols SA (NASDAQ:GRFS), a global healthcare company, said it plans to initiate a safety trial later this year of a new inhaled formulation for treating emphysema due to alpha1-proteinase inhibitor deficiency.
The Barcelona, Spain-based company said it would initiate a safety trial of a novel, inhaled formulation of alpha1-proteinase inhibitor (Human), following orphan drug designation for cystic fibrosis.
Grifols currently leads the market in alpha1-proteinase inhibitors with its 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 alpha 1-proteinase inhibitor as an inhaled formulation.
PROLASTIN-C, Alpha1-Proteinase Inhibitor (Human) is indicated for chronic augmentation and maintenance therapy in adults with emphysema due to deficiency of alpha1-proteinase inhibitor (alpha1-antitrypsin deficiency).
The effect of augmentation therapy with any alpha1-proteinase inhibitor (alpha1-PI) on pulmonary exacerbations and on the progression of emphysema in alpha1-antitrypsin deficiency has not been demonstrated in randomized, controlled clinical trials.
In April, the U.S. Food and Drug Administration (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.
Alpha 1-antitrypsin deficiency is a genetic disorder that causes defective production of alpha 1-antitrypsin (A1AT), leading to decreased A1AT activity in the blood and lungs, and deposition of excessive abnormal A1AT protein in liver cells. Severe A1AT deficiency causes panacinar emphysema or COPD in adult life.
GRFS is trading up 2.64% at $10.30 on Tuesday. The stock has been trading between $4.45 and $10.31 for the past 52 weeks.