Developing Alpha-1 Antitrypsin Therapeutics; Public Workshop

Developing Alpha-1 Antitrypsin Therapeutics; Public Workshop

The Food and Drug Administration (FDA) is announcing the following public workshop entitled “Developing Alpha-1 Antitrypsin Therapeutics.”


Purpose of the workshop:


The purpose of the public workshop is to foster the development of therapeutic products for the treatment of alpha-1 antitrypsin deficiency (AAT deficiency).




AAT deficiency is a hereditary disorder that can be associated with the development of lung and liver disease. The age at which individuals initially present with the signs and symptoms of AAT deficiency can vary, but individuals often present between 20 and 50 years of age. Symptoms of lung disease are more common than liver disease and may include wheezing, shortness of breath with activity, and reduced exercise tolerance. Ultimately, emphysema (the state of irreversible damage to and partial loss of the small air sacs of the lung) may occur. The symptoms of liver disease may include yellowing of the skin and the whites of the eyes (jaundice), and, if more advanced, swelling of the abdomen due to cirrhosis (liver damage).


Symptomatic treatments, including medications called bronchodilators and oxygen, may be given for the lung disease in AAT deficiency, and in severe cases lung transplantation may be considered. Currently, no specific therapy for AATD-related liver disease is available other than liver transplantation if liver failure occurs. AAT augmentation therapy through the administration of this enzyme inhibitor (Alpha-Proteinase Inhibitor (Human)) prepared from human plasma may be used in those with AAT-related lung disease. Although research suggests that this therapy may be helpful, there is some uncertainty as to the extent of its effectiveness. New therapies, such as gene replacement, are currently in development to try to address the various manifestations of AAT deficiency.


Topics for Discussion at the Public Workshop:


The workshop will include presentations and a panel discussion by experts from academic institutions, industry, and government agencies on the following topics: (1) Background on AAT deficiency and augmentation therapy, including safety and efficacy of these products; (2) the perspective of patients with AAT deficiency; (3) approaches to the design of clinical trials in individuals with AAT deficiency; (4) potential outcome measures for use in clinical trials; (5) therapeutic approaches to AAT deficiency, including novel therapeutic approaches and therapeutic targets; (6) products in development; and (7) a panel discussion on the current needs in AAT drug development.




September 16, 2019

8:00 AM – 5:00 PM




Natcher Conference Center

Building 45

National Institutes of Health Campus

9000 Rockville Pike

Bethesda, MD 20892


Entrance for the workshop is through Building 66 (Gateway Center), where routine security check procedures will be performed. For parking and security information, please refer to  



Registration: To register for the public workshop, please visit the following website:


Please provide complete contact information for each attendee, including name, title, affiliation, and email address. Registration is free and based on space availability, with priority given to early registrants. Persons interested in attending this public workshop must register by September 3, 2019, midnight Eastern Daylight Time. Early registration is recommended because seating is limited. If time and space permit, on-site registration on the day of the public workshop will be provided, beginning at 7 a.m. If you need special accommodations due to a disability, please contact Loni Warren Henderson or Sherri Revell no later than September 9, 2019.




Loni Warren Henderson or Sherri Revell, Center for Biologics Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, MD 20993, 240-402-8010, email (subject line: Alpha-1 Workshop).