Hereditary Angioedema Drug Shows Safety and Efficacy in Pediatric Patients, Study Shows

According to a story from Angioedema News, a recent clinical trial has demonstrated that the drug conestat alfa (marketed as Ruconest) was able to successfully treat 96 percent of acute swelling episodes in children with the rare genetic disorder hereditary angioedema. The drug was approved as a treatment for the disease in Europe in 2010 and was approved in the US in 2014. However, its effects in child patients were not backed by strong data.

About Hereditary Angioedema

Hereditary angioedema is a genetic disorder which is characterized by chronic episodes of swelling that can affect multiple areas of the body. The condition is caused by mutations affecting the HAE gene. Swelling attacks generally occur every two weeks or so; they can usually last for several days. Swelling may affect the limbs, digestive tract, face, and airway, with blockage of the airway being the most dangerous complication. Vomiting and abdominal pain may accompany attack as well if the digestive tract is involved. Treatment involves reducing the likelihood for attacks to appear and preventing them from worsening when they do. Hereditary angioedema is typically only life-threatening if left untreated. Prevalence of the condition is estimated to be around one in 10,000 to one in 50,000, at least in the US and Canada. To learn more about hereditary angioedema, click here.

About Conestat Alfa

Conestat alfa works by replacing a critical C1 inhibitor protein that, in patients, is deficient or absent as a result of genetic mutation. Symptoms of swelling are triggered by a lack of this protein in the blood; therefore, supplementing that protein with conestat alfa can relieve swelling.

Study Results

The trial included a total of 20 child hereditary angioedema patients that were between the ages of five and 13. The patients were to receive an intravenous dose of conestat alfa in response to acute swelling episodes as they appeared. The dose level was 50 U/kg. The drug was used to treat a total of 73 attacks during the study. The primary endpoint was the duration from the time the attack appeared to the start of symptom relief. A secondary endpoint was time to minimal symptoms.

The drug was able to halt 95.9 percent of attacks. Over a third of the participants required treatment for four or more episodes. The median time to symptom relief was 60 minutes and the median time to minimal symptoms was 122.5 minutes.

This study clearly demonstrated that this drug was effective in this patient population with no serious safety concerns. Check out the original study here.


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