Featured Research

from universities, journals, and other organizations

New drug shown safe, effective in treating hereditary angioedema

Date:
August 6, 2010
Source:
Massachusetts General Hospital
Summary:
Clinical trials from two international research teams have shown that icatibant, a new drug that blocks the action of an inflammatory protein known as bradykinin, is safe and effective in treating acute attacks of hereditary angioedema, a potentially life-threatening condition.

Clinical trials from two international research teams have shown that icatibant, a new drug that blocks the action of an inflammatory protein known as bradykinin, is safe and effective in treating acute attacks of hereditary angioedema, a potentially life-threatening condition.

Related Articles


In their report in the August 5 New England Journal of Medicine, the authors note that -- while the results of one trial did not reach statistical significance -- the drug is safe and effective and further study will help clarify the patients and symptoms best treated with icatibant.

"We have not had many options for treating painful, debilitating and potentially life-threatenting attacks of hereditary angioedema, and these studies showed that icatibant improves symptoms and is not associated with any concerning side effects," explains Aleena Banerji, MD, of the Massachusetts General Hospital (MGH) Rheumatology, Allergy and Immunology Division, corresponding and co-lead author of the NEJM paper, who also was principal investigator of the MGH study site.

Hereditary angioedema (HAE) is caused by low levels or poor function of a protein called C1 esterase inhibitor. Patients with the condition suffer recurrent episodes of swelling caused by fluid leaking from blood vessels. The swelling can involve the face, extremities, gastrointestinal tract and the throat or larynx, where it can cause potentially life-threatening airway blockage. HAE is believed to affect at least one in 50,000 people, and treatment has been focused on preventing attacks and relieving symptoms. The FDA has approved C1 esterase inhibitor infusions for treatment of HAE, and an injected drug that inhibits the enzyme kallikrein also recently recieved approval to treat acute HAE attacks. But that approval stipulated the treatment could only be administered by a health care professional because of the risk of anaphylactic reactions.

Icatibant blocks the receptor for bradykinin, a protein that dilates and increases the permeability of blood vessels and produces many symptoms of inflammation. Bradykinin is believed to mediate many symptom of HAE, so a drug that interferes with its action was felt to be a good candidate to treat HAE attacks. Manufactured by the German pharmaceutical firm Jerini, Inc., icatibant has received approval in the European Union, where it is marketed under the brand name Firazyr. The NEJM study reports on two phase 3 trials -- one based in the U.S. the other in Europe -- conducted as part of the Jerini's application to the U.S. Food and Drug Administration.

Both studies were randomized, double-blind, prospective trials enrolling adults who had been diagnosed with HAE. The U.S.-based trial, called FAST-1, compared icatibant with a placebo for treatment of acute attacks; while the European trial, FAST-2, used trenaxminic acid, an oral medication available in Europe, as the comparison drug. Study participants -- 56 for FAST-1 and 74 for FAST-2 -- returned to their enrollment site for treatment within six hours of onset of a moderate to severe HAE attack. Response to the study medication was assessed by both patients themselves and by study investigators, who primarily evaluated the time required for relief of the most severe symptom and secondarily evaluated relief of all symptoms.

In both studies, icatibant produced faster symptom relief than the comparison drug, but the difference was much greater in the FAST-2 trial that used trenaxminic acid for comparison. The authors note that the less significant results seen in FAST-1 may reflect aspects of the study design -- particularly the fact that success was determined based on relief of the most serious symptom only. The limited size of the study groups means that they may have differed in the primary symptoms patients experienced. In addition, more FAST-1 participants received C1 esterase inhibitor injections as rescue treatment, which may have hidden the extent of icatibant-associated symptom improvement. No serious treatment-related adverse events were reported in either trial.

"These data do show that HAE patients receiving icatibant improve, and the drug seems to be very safe," says Banerji. "A larger, worldwide phase 3 trial of icatibant is currently in process and should help us clarify the picture further." Banerji is an assistant professor of Medicine at Harvard Medical School.

The NEJM paper was co-authored by investigators from centers in 15 countries, including Mario Cicardi, MD, University of Milan, co-lead author with Banerji and principal investigator of the Milan study site; and Kimberly Rosen, MD, and Wing-Tse Fan, PhD, of Jerini, Inc. The company wholly funded the FAST-2 trial and, along with the U.S. National Institutes of Health, partially funded FAST-1. While the company designed both trials, data monitoring and collection were handled by outside agencies, and the academic co-authors were responsible for analyzing and reporting study results.


Story Source:

The above story is based on materials provided by Massachusetts General Hospital. Note: Materials may be edited for content and length.


Journal Reference:

  1. Cicardi et al. Icatibant, a New Bradykinin-Receptor Antagonist, in Hereditary Angioedema. New England Journal of Medicine, 2010; 363: 532-541 DOI: 10.1056/NEJMoa0906393

Cite This Page:

Massachusetts General Hospital. "New drug shown safe, effective in treating hereditary angioedema." ScienceDaily. ScienceDaily, 6 August 2010. <www.sciencedaily.com/releases/2010/08/100804205143.htm>.
Massachusetts General Hospital. (2010, August 6). New drug shown safe, effective in treating hereditary angioedema. ScienceDaily. Retrieved November 26, 2014 from www.sciencedaily.com/releases/2010/08/100804205143.htm
Massachusetts General Hospital. "New drug shown safe, effective in treating hereditary angioedema." ScienceDaily. www.sciencedaily.com/releases/2010/08/100804205143.htm (accessed November 26, 2014).

Share This


More From ScienceDaily



More Health & Medicine News

Wednesday, November 26, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

From Popcorn To Vending Snacks: FDA Ups Calorie Count Rules

From Popcorn To Vending Snacks: FDA Ups Calorie Count Rules

Newsy (Nov. 25, 2014) — The US FDA is announcing new calorie rules on Tuesday that will require everywhere from theaters to vending machines to include calorie counts. Video provided by Newsy
Powered by NewsLook.com
Daily Serving Of Yogurt Could Reduce Risk Of Type 2 Diabetes

Daily Serving Of Yogurt Could Reduce Risk Of Type 2 Diabetes

Newsy (Nov. 25, 2014) — Need another reason to eat yogurt every day? Researchers now say it could reduce a person's risk of developing type 2 diabetes. Video provided by Newsy
Powered by NewsLook.com
Madagascar Working to Contain Plague Outbreak

Madagascar Working to Contain Plague Outbreak

AFP (Nov. 24, 2014) — Madagascar said Monday it is trying to contain an outbreak of plague -- similar to the Black Death that swept Medieval Europe -- that has killed 40 people and is spreading to the capital Antananarivo. Duration: 00:42 Video provided by AFP
Powered by NewsLook.com
Are Female Bosses More Likely To Be Depressed?

Are Female Bosses More Likely To Be Depressed?

Newsy (Nov. 24, 2014) — A new study links greater authority with increased depressive symptoms among women in the workplace. Video provided by Newsy
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
 
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:  

Breaking News:

Strange & Offbeat Stories

 

Health & Medicine

Mind & Brain

Living & Well

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:  

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile iPhone Android Web
Follow Facebook Twitter Google+
Subscribe RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins