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Please Participate in HAE Clinical Trials and Fill Out the HAE Association's Clinical Trial Interest Form
Currently there are four companies conducting clinical trials with the goal of getting their HAE treatments. Rare diseases and small patient populations usually do not receive this much attention, so we must take full advantage of this "once in a lifetime opportunity" for approval of several different HAE therapies. However, nothing will happen unless we make sure each of these trials has sufficient participants. We hope each and every HAE friend will help themselves and other family members by participating.
HAE patients interested in clinical trials can either fill out our Clinical Trial Form, or contact The HAE Association's Clinical Trial Programs Director Donna Davis.
Please note: Any information you provide to the HAE Association is carefully safeguarded to eliminate even the slight possibility of unauthorized disclosure.Once you have completed the form, Donna Davis or one of her Clinical Associates will contact you with information regarding clinical trial sites in your area. Alternatively, if you have frequent attacks and indicate that you are willing to travel and stay in a hotel near a trial site (travel and lodging expenses will be paid by the trial sponsor), you will be contacted with information regarding how this could be arranged. It is important to note that the HAE Association will work with you, your doctor, and the pharmaceutical company to make sure you have medicine available so you can get treated for attacks that occur after your travel is completed.
Below is some information regarding the companies looking to license their medicines in the United States
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ViroPharma (formerly known as Lev Pharma) ViroPharma's C1 inhibitor (the brand name is Cinrzye) was approved by the FDA as an attack prevention therapy and is now commercially available in the US. Cinryze is currently under priority review by the FDA for acute attack prevention. The FDA is expected to make a decision in June 2009.
ViroPharma has put together an infrastructure to make Cinryze available to every HAE friend that could benefit from the medicine. The program is called Cinryze Solutions, and the HAE Association is ready to (1) provide you with a direct referral into the Cinryze Solutions program, and (2) act as your advocate and confidant and help you work with your doctor, the specialty pharmacy who will ship your medicine, drug company representatives, and insurance providers. Please contact the HAEA's Patient Services group for more information-- Donna Davis donna-davis@haea.org or Michelle Williamson michellewilliamson@haea.org. - CSL Behring (Berinert brand of C1 inhibitor) has completed its phase 3 trial and expects Berinert to be approved for treating acute HAE attacks (attacks that have begun) sometime in 2009. CSL Behring officials noted that the company is a global leader in the plasma protein biotherapeutics industry, and are experts in producing products to treat rare diseases, and that Berinert has been safely and effectively used throughout the entire world for decades.
- Dyax (Ecallantide) completed its second phase 3 clinical trial for its subcutaneous HAE attack treatment and reported that the data generated during the trial indicates the medicine is an effective and safe therapy. Ecallantide is a recombinant product that inhibits kallikrein-an enzyme that plays a key role in the biochemical processes that lead to HAE-related swelling. The company filed for FDA licensure in September 2008, and the product could be approved in early 2009.
- Jerini AG (Firazyr) conducted a US clinical trial that did not meet the statistical significance requirements for effectiveness required by the FDA. Firazyr is delivered through a subcutaneous injection and works by blocking the peptide that causes HAE-related swelling. The product has been licensed in Europe and Jerini is now working on plans to conduct another clinical trial in the US.
- Pharming NV (recombinant C1 inhibitor) announced successful clinical trial results for their product, Rhucin-a recombinant human C1 inhibitor protein that is derived from the milk of genetically altered rabbits. Clinical trials have shown that Rhucin is a safe and effective treatment for acute HAE attacks. The company plans to apply for a US license in the near future.
