CHMP recommends approval of Respreeza (alpha1-antitrypsin), for alpha-1 antitrypsin deficiency- CSL Behring

The European Medicines Agency Committee for Medicinal Products for Human Use (CHMP) has recommended granting marketing authorization for Respreeza (alpha1-antitrypsin), from CSL Behring, indicated to treat patients with alpha-1 antitrypsin deficiency (AATD). AATD is a hereditary condition marked by a lack of the alpha-1 antitrypsin protein, whose main function is to protect the lungs from inflammation. Respreeza replaces the protein that these patients are missing and raises the alpha-1 antitrypsin levels in their blood, which can help to protect the lungs from damage due to inflammation. The drug was approved in 2012 by the FDA under the name Zemaira.

CSL Behring's RAPID (randomized, placebo-controlled trial of augmentation therapy in alpha-1 proteinase inhibitor deficiency) study results were considered as part of this submission. According to findings of the study, patients with AATD treated with alpha-1 proteinase inhibitor therapy exhibited a lower annual rate of lung density decline compared to placebo, when measured using chest computed tomography, at full inspiration. This demonstrated that Respreeza significantly slows the progression of emphysema in these critically ill patients.