Beth Mercante'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 Beth Mercante. I am here today thanks to travel provided by the HAE Association. I have no financial ties to LEV whatsoever. I am here to provide the committee with some insight into the life altering impact of the Lev Pharma C1 inhibitor product.

I have experienced overwhelmingly positive results by participating in the Lev prophylaxis trial for over two years. I am one of the 20 patients, Dr. Kalfus and Dr. Busse mentioned in their presentation, who continue to do remarkably well in the open label extension. Two infusions per week at the clinic are a small inconvenience for being able to have a virtually attack free life.

While, I am not here today to focus on me, I think the committee should know that before I gained access to Lev’s C-1 inhibitor, I missed countless school, work, and childcare days because HAE swelling left me either grossly disfigured, doubled over with excruciating abdominal pain, or wondering if the throat attack underway would be the one to finally take my life.

Instead, I am here today because I have a 7-year-old son Julian, who has HAE, and I am compelled to do whatever I can to make sure that C1 inhibitor is approved so he does not have to experience the pain and suffering that my family members and I have endured in our lifetimes.

One morning, when my son was 18 months old, I went to get him out of his crib and saw something that changed my world forever.

My precious child was beaming at me with usual loving grin, but his eyes were almost swollen shut, and his lips were about 5 times their normal size.

I immediately knew that he has inherited my genetic deficiency and had HAE.

Before Julian was accepted for open label acute treatment with the Lev C1 inhibitor product. He experienced horrific abdominal attacks that resulted in a level of suffering that was unbearable to witness.

My family represents both sides of the spectrum with regard to the therapy needed by HAE patients.

I am doing beautifully on prophylaxis, and access to acute therapy has radically reduced my son’s HAE-related suffering.

One morning a few months ago, Julian was getting ready for school and I noticed that he was walking slowly and gingerly.

As an HAE patient, I immediately knew what was going on and said, “You look like your tummy is really swollen, do you want to stay home from school?”

He replied, “No mommy, I don’t want to miss school, I think I will be ok.”

Coincidentally, I had a meeting at his school that morning, so when it ended I headed off to Julian’s classroom to see how he was doing.

As I walked down the corridor, I saw my son’s second grade class coming down the stairs and I ran over to see if Julian was feeling better.

At first, my son was nowhere to be seen, but a few moments later; there he was, shuffling along in obvious pain, barely capable of walking down the stairs.

I scooped him up into my arms and told him it was time to make him feel better.

We headed off to the Lev clinical trial center where the C1inhibitor was waiting for us.

Twenty minutes post infusion, Julian was able to walk normally; and within forty minutes, he was doing jumping jacks in the waiting room to show how much better he was feeling.

I had tears of joy in my eyes as I thanked the compassionate doctors and nurses for all they are doing to treat this debilitating and deadly disease.

Based on experience, I can look each and every member of the committee and FDA staff directly in the eye and tell you that the LEV C1 inhibitor product is remarkably safe.

Both prophylactic and acute therapies are vital and offer the prospect of a normal life for me, my son and thousands of other patients just like us.

Too many of us have suffered for far too long---both prophylactic and acute therapies deserve swift approval!!

Thank you.