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Print this pageMay 2, 2008 - An Important Milestone for HAE Patients
Dear HAE Friends,Today is a great day for US HAE patients! I just got back from a meeting of the FDA's Blood Products Advisory Committee which recommended--in a stunning 21-0 vote--that the FDA approve the Lev Pharma C1 inhibitor product for "preventing" HAE attacks.
Special thanks are due to the HAE Association's Director of Operations, Dennis DeMarinis, for taking care of all the essential details that made today's appearance possible and successful.
The HAE Association had a contingent of 5 patients and a physician who spoke before the Committee and the FDA staff. Your fellow HAE friends--Janet Long, Beth Mercante, Michelle Williamson, Tracy Conaway and Sally Urbaniak (along with some incredibly on-point remarks by Dr. Amin Barakat who treats my severely affected children) were not only notably professional and articulate, but had a tremendous impact on the Committee's deliberations! The intelligence and passion that our patients brought to the proceedings was nothing short of remarkable (copies of their testimonies are available at the bottom of this page).
Experts who work with the FDA and Advisory Committees warned us that It is very difficult to get the attention of physicians and scientists who usually only care about esoteric statistical analysis. Well, your HAE friends succeeded in proving the experts wrong! The speeches made by HAE friends had a remarkable impact on today's proceedings. It was amazing to see both the Blood Product Advisory Committee and the FDA staff pay rapt attention to every word that HAE patients offered. When we were done, the tone of discussion changed dramatically. Taking the lead from our comments, the Committee began asking the FDA staff pointed questions about why treatment for attacks that are underway (acute attacks) was not being considered. This was a huge victory for our patient community, and the Committee made it very clear that the FDA staff needed to expedite their review of the data gathered during the acute attack treatment clinical trial as well.
The Blood Products Advisory Committee's unanimous endorsement of licensing C1 inhibitor in the United States is a huge first step towards our shared goal of providing all HAE friends with access to a safe and effective non steroidal HAE therapy. We have won our first important battle in this 6 year struggle, and with the help of each and every HAE friend in our community, we are going to win this war!
We will keep you informed on developments as they unfold.
Warm regards to all,
Anthony J. Castaldo
President, US HAE Association
Anthony Castaldo's testimony
Sally Urbanek's's testimony
Janet Long's testimony
Michelle Williamson's testimony
Dr. Barakat's testimony
Beth Mercante's testimony
Tracy Conaway
