HAE Clinical Trials Offer Hope for Better Therapeutic Alternatives
Five companies are currently conducting or have recently concluded their HAE clinical trials. Below we summarize the status of each company's clinical program.
VIROPHARMA-C1 Inhibitor Concentrate (double nanofiltered)
In Oct 2008, ViroPharma received FDA approval for Cinryze, the company's C1 inhibitor concentrate product, as an attack prevention therapy. The European brand of this product has an impressive safety record spanning three decades; and the version that will be sold in the US offers enhanced assurance by adding double nano filtration.
Cinryze is now commercially available. ViroPharma has put together an infrastructure, Cinryze Solutions, to make Cinryze available to every HAE patient that could benefit from the medicine. Please contact the HAEA's Patient Services group for more information-- Donna Davis donna-davis@haea.org or Michelle Williamson michellewilliamson@haea.org.
Cinryze is currently under priority review by the FDA for acute attack prevention. The FDA is expected to make a decision in June 2009.
CSL BEHRING-C1 Inhibitor Concentrate
The Berinert ™ brand of C1-inhibitor concentrate was FDA approved on October 9, 2009. The product label states that Berinert is indicated for acute facial and abdominal HAE attacks. Berinert ™ is delivered intravenously and will be available in the United States in December 2009.
DYAX CORP-Kallikrein Inhibitor
Kalbitor®, from Dyax Corp., was approved in December 2009 to treat acute HAE attacks in patients 16 years of age and older. The product is delivered through subcutaneous injections and is estimated to be available in the US in early 2010.
JERINI AG-B2 Bradikynin Receptor Antagonist
The German biotechnology company Jerini AG has completed a US clinical trial of their B2 bradykinin receptor antagonist--Icatibant. Jerini reported that although the results of this trial were clinically relevant, the focus of the study--median time to onset of symptom relief of drug versus that of placebo--did not reach statistical significance. A separate concurrent study conducted mostly in Europe did show a statistically significant difference between Icatibant and Tranexamic Acid (which is used as an acute therapy by some European doctors). Since then, 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
The Dutch biotechnology company, Pharming NV is testing their recombinant C1 inhibitor product in the US. Pharming's product is derived from the milk of female rabbits that have been genetically altered to produce the human C1 inhibitor protein. 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. Pharming is currently still accepting patients into its clinical trials. Click here to sign up for a clinical trial.
