Michelle Williamson'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 Michelle Williamson. I do not have any financial ties to Lev Pharma. I am not a shareholder. I work for the HAE Association. I am one of the hundreds, if not thousands of HAE patients for whom 17 alpha alkylated androgens are not effective.

During 23 long years of androgen therapy, I suffered through countless emergency room visits for attacks that not only involved excruciating abdominal pain, but also involved my airway and required more than a dozen laryngeal intubations and an emergency tracheotomy.

I was disabled for the majority of my adult life, but access to C1 inhibitor through Lev Pharma’s open label program allowed me to finish my college degree.

I have held a job now for well over a year.

I am a living, breathing example of why HAE patients in the United States desperately need C1 inhibitor-- but not only for preventing HAE attacks. Life saving acute therapy is every bit--if not more--important and I truly hope the Committee understands that throat attacks and excruciating abdominal attacks can occur at any time; even for patients on androgen or C1INH prophylaxis. A tragedy that almost took my life last February illustrates this point.

After receiving open label C1 inhibitor therapy for a number of months, I felt joy for the first time in well over a dozen years and decided I was well enough to take an out of town weekend trip. I had what can only be described as an idyllic “getaway” until HAE cruelly asserted itself. While on the way to catch a return flight home, I realized I was experiencing a laryngeal attack and it was coming on rapidly.

My boyfriend noticed that I was having trouble swallowing so he flagged a police officer who called an ambulance. At the ER--despite my objections--the doctors treated me with medicines HAE patients know do not work--epinephrine and Benadryl. They also tried Fresh Frozen Plasma to no avail.

The advance of the swelling attack and the baffled look on the faces of the ER staff made me fear for my life, and I prepared myself to die – again. I told my boyfriend to tell my son that I loved him, was proud of him, and that I was sorry and to call my mom and my sisters. As I lay helpless with my airway tightening, I remember coughing and then, nothing else.

I spent the next 7 days intubated and sedated. My lungs had collapsed and I lost the use of my leg muscles from being bedridden. I could barely manage to sit up until day 11 when I managed to take three steps before hyperventilating and fainting.

I woke up hearing doctors trying to decide whether or not to intubate again and telling me I should consider a permanent tracheotomy.

After 19 days in the hospital and an 80 thousand dollar bill, I was sent home with antibiotics to treat hospital-acquired pneumonia and endured weeks of rebuilding leg muscles so I could walk again.

The tragedy is that this entire situation would have been avoided by two vials of C1 INH concentrate!

As you deliberate approving the Lev Pharma product today, I kindly ask you to consider patients like me who need this therapy for both preventing and treating dangerous acute HAE attacks.

Thank you.