Dr. Amin Barakat's Testimony Before the FDA's Blood Products Advisory Committee - May 2 2008

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

Good Morning. My name is Dr. Amin Barakat. I am Clinical Professor of Pediatrics at Georgetown University Medical Center. I also practice pediatrics in Northern Virginia.

I am here today to provide the committee and the FDA staff with the viewpoint of a physician who witnessed the severity of the HAE disease and who supervised a severely affected patient on open label prophylaxis therapy with Lev Pharma’s C1 Esterase Inhibitor product.

I personally witnessed how access to, and the prudent use of this remarkable medication transformed a disabled young woman into a healthy, vibrant and successful person. This medication provided the most dramatic outcome I have witnessed in over 35 years of pediatric practice.

I am troubled that the committee is considering approval of C1 Esterase Inhibitor for prophylaxis only, especially since the acute trial has also met its clinical endpoint. I would like to discuss an incident that provides compelling evidence that prophylaxis alone does not provide HAE patients with the full range of therapy needed to treat this unpredictable, life threatening disease.

My patient was working on a college project that required out of town travel. Since this young lady often experiences dangerous laryngeal swelling, we worked things out to make sure she would be back in town for her regular infusion. Unfortunately, the return flight that would have allowed her to keep her prophylaxis schedule was cancelled. The patient got on the next morning flight, but right before landing, she began experiencing an acute attack. Upon deplaning, she called my office and said she was very sick and was on her way for a treatment.

I examined her as soon as she arrived and noted severe abdominal pain, dehydration and hypotension. Fortunately, our nurse had already prepared the C1 Esterase Inhibitor but dehydration made it difficult to find a vein and it took two of us to start the infusion.

It was very evident to me then that any further deterioration in the patient’s condition would warrant hospitalization and I told the patient that I might have to send her to hospital by ambulance.

Despite being severely ill, the young lady was adamant that she would get better quickly, and sited past use of imported C1 Esterase Inhibitor that provided rapid and dramatic relief from severe acute attacks.

I continued to monitor her vital signs and noted marked improvement after 15 minutes of treatment. I was astounded that 40 minutes post infusion, the youngster’s vital signs were normal, and she was demanding that we allow her to leave so she could attend her late afternoon class. Without access to C1 Esterase Inhibitor, the acute attack would have required hospitalization, and this young lady would have had to suffer for 48-72 hours.

My patient’s experience illustrates that the HAE patient community would be ill served by an approval that ignores the important need and life saving value of acute C1 Esterase Inhibitor therapy.

Thank you.