The liquid alpha-1 proteinase inhibitor is intended to treat alpha-1 antitrypsin deficiency.

Children born with the inherited disease alpha-1 disorder alpha-1 antitrypsin deficiency (ATDD) often suffer a progressive loss of pulmonary function. Unfortunately, AATD, which causes a deficiency or absence of alpha-1 protein in a patient’s plasma, occurs at a higher rate than other rare lung diseases such as cystic fibrosis and pulmonary arterial hypertension, affecting approximately 25 out of 100,000 people, according to the Orphanet Report Series in the Rare Diseases Collection, which was published in May 2014. Most of these people — more than 90% in fact, remain undiagnosed.

In total, approximately 100,000 people each in the US and Europe are thought to suffer from AATD, which carries symptoms including shortness of breath (dyspnea) following physical exertion; chronic coughing; excessive mucous production; and wheezing, all with or without the presence of respiratory infections. Because these symptoms are similar to those of other pulmonary diseases such as chronic obstructive pulmonary disease (COPD) and asthma, many patients do receive treatment for their symptoms, but do not address the cause of their problems. This situation is complicated by the fact that early diagnosis of AATD is necessary to reduce the risk of patients developing pulmonary emphysema, which can prove fatal without a lung transplant. In children, early treatment is essential to prevent liver disease, which is most commonly caused by AATD.

AATD patients in the US now have a new option for their treatment following U.S. Food and Drug Administration (FDA) approval of Prolastin^®-C Liquid from Grifols, the first liquid formulation of an alpha-1 antitrypsin deficiency replacement therapy manufactured in the U.S, according to the company. The Grifols liquid treatment is advantageous compared to an existing lyophilized drug product because it is ready to infuse and requires less preparation time. Compared to a different product, it requires a lower volume of liquid for infusion.

FDA approval of the new product represents an achievement of an important R&D milestone for Grifols with regard to creating a new formulation of alpha-1 antitrypsin. The commercial launch of Prolastin^®-C Liquid in the US is scheduled for 2018.

The company has also submitted a new drug application for its liquid alpha-1 proteinase inhibitor with the European Medicines Agency (EMA). Meanwhile, Grifols recently received a positive recommendation from the EMA’s Committee for Medicinal Products for Human Use (CHMP) for VeraSeal^® (human fibrinogen/thrombin), a new product used for surgical operations in adults.