HAE Association'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

"My name is Anthony Castaldo and I am the President of the United States Hereditary Angioedema association and also the president of Hereditary Angioedema International.

I thank you for providing HAE patients with an opportunity to discuss the critical need for a safe and effective non steroidal HAE therapy. I do not have any financial ties to Lev Pharma and I am not a shareholder.

Perhaps the best characterization of how HAE affects patients appeared in a 1996 New England Journal of Medicine article:

"Patients with a deficiency of c1 inhibitor are not just an interesting model for study.... They are critically ill, and many have ancestors who died suddenly from suffocation. Patients live in constant dread of life-threatening laryngeal obstruction."

It is both ironic and sad that the very same New England Journal Of Medicine volume –published 12 long years ago--also contained a paper authored by Dr. Frank, (who is here today) that summarized the results of a double blind placebo controlled cross-over study that demonstrated the safety and statistically significant efficacy of a C1 inhibitor concentrate product.

Ladies and gentleman, Hereditary Angioedema represents a catastrophic unmet medical need in the United States. While 17 alpha alkylated anabolic steroids are useful for HAE prophylaxis in certain adults, the scientific literature reveals that many patients continue to experience periodic acute abdominal and laryngeal attacks notwithstanding ongoing therapy. Moreover, the utility of these agents is limited because they are not well tolerated by women, are directly linked to increased serum lipid levels, and their use is contraindicated for children, many of whom, tragically, are severely affected and suffer frequent attacks.

Is it not paradoxical that 350 lb NFL linemen are suspended and counseled on the extreme danger, toxicity, and undesirable long term side effects posed by relatively short courses of the same drugs that our patient community is forced to use on a chronic basis.

We are delighted that the Committee will be considering the prophylaxis indication today. To be sure, prophylaxis will serve to finally ameliorate HAE’s dreadful morbidity. Nevertheless, prophylaxis will only address a portion of the problem faced by HAE patients. As noted earlier, the scientific literature provides ample evidence that patients can have break through life threatening laryngeal or excruciating abdominal attacks no matter what therapy they are using. Keep in mind that HAE is a very unpredictable disease.

Why should a patient who only has one attack a month or less be forced to take androgens every day when documented experience from hundreds of patients in Germany, Denmark, and the Netherlands demonstrates that less severely affected patients prefer to manage their HAE by working with their physicians to set up on-demand access for acute attacks.

You have before you today a preponderance of evidence demonstrating that the Lev Pharma C1 inhibitor product is both safe and effective. We hope that the committee recognizes that EVERYONE in our beleaguered patient community deserves the opportunity for access to a non steroidal alternative that provides relief from this catastrophic unmet medical need.