Janet Long'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
Janet Long's testimony
Dr. Barakat's testimony
Beth Mercante's testimony
Tracy Conaway

Good morning. My name is Janet Long. I do not have any financial ties to Lev Pharma, I am not a shareholder and the HAE Association paid for my travel here today. I would like to provide the Committee and FDA staff with the perspective of a typical HAE patient who endured countless years of suffering before getting a proper diagnosis. Unfortunately, I do not live close enough to participate at a Lev Pharma clinical trial site, so I must rely on androgens to maintain a semblance of normal life.

I would like to begin my remarks by providing a specific case history that illustrates why HAE is a catastrophic unmet medical need in the United States.

I began having severe abdominal attacks at age seven and to this day I am haunted by the horrible look of helplessness on my mother’s face when she could only offer me a hot water bottle and a couple of baby aspirins; treatments that she knew would do nothing to ease my suffering.

At age 21, I experienced an abdominal episode that was so severe it caused bleeding which led to an unnecessary exploratory laporotomy and days in the Intensive Care Unit. Despite this catastrophic event, no one could figure out what was wrong with me and the ensuing years brought nothing but ceaseless agony, including laryngeal attacks that came close to completely closing my airway. I saw scores of doctors who either admitted to being totally baffled or offered diagnostic theories that, at least to me, never seemed to make much sense.

Over the years, I continued to suffer and I felt hopeless, caught in the grips of a mysterious illness that no one, including me, understood. I had tired of showing up at the Emergency Room only to be sent home after being told that nothing could be done, and that I would have to learn to live with my condition.

In light of these distressing experiences, I chose to endure the excruciating pain and discomfort associated with abdominal attacks at home where at least I was in familiar surroundings. In retrospect, I realize that might not have been the best idea, but at the time I felt trapped and, again, hopeless and I resigned myself to an unknown fate.

I remember vividly one particularly devastating attack that had me curled up in agony - the pain and nausea had become so unbearable I was convinced I was going to die. I faced what I believed was the very real possibility that my three beautiful young daughters would be left motherless, and through my tears, I told my husband, "If I don't make it through the night, please tell the girls that I loved them."

Finally, after almost forty years of horrific suffering with the prospect of living getting bleaker as the attacks continued unabated, a brilliant gastroenterologist unraveled the mystery and came up with the diagnosis of Hereditary Angioedema. I have been on androgens ever since and daily endure the unfortunate and embarrassing side effects one would expect for a woman on long-term therapy with a potent male hormone.

Today, you have before you two separate double blind placebo controlled studies that provide statistically significant evidence of something European doctors and patients have known for over three decades—C1 inhibitor concentrate has amassed a remarkable safety record and is the unequivocal gold standard treatment for Hereditary Angioedema.

This Committee’s recommendation for approval and swift action by the FDA staff will prevent other mothers from the fear of leaving their children behind and ensure that no one else has to endure 40 years of lonely and horrifying pain. The evidence is compelling, the need is overwhelming, and any further delay in helping this underserved patient community would be tragic.

Thank you for allowing me to address you today.