The U.S. Food and Drug Administration approved Haegarda, the first C1 Esterase Inhibitor (Human) for subcutaneous administration to prevent Hereditary Angioedema (HAE) attacks in adolescent and adult patients.
Current treatment of HAE is an injection of C1 esterase inhibitor into veins. This route injects the medication just under the skin, allowing for easier self-injection by the patient or caregiver.
HAE is a rare, genetic, and potentially life-threatening condition. It causes painful, debilitating, and unpredictable episodes of abdominal pain and swelling of the face, extremities, and the airways.
Attacks can occur spontaneously, or can be triggered by stress, surgery or infection.
Depending on the severity of the disease, patients could experience several debilitating attacks each month. More than half of all HAE patients experience laryngeal attacks during their lifetime, which block the airway and can be fatal. Mortality rates are as high as 33 percent in untreated patients.
The disease is caused by deficient or dysfunctional C1-INH, a protein in the blood that helps to control inflammation. According to CSL Behring, manufacturer of Haergarda, the drug works by replacing deficient or dysfunctional C1-INH protein, restoring C1-INH levels above 40 percent of normal levels, which is associated with reduced risk of HAE attacks.
Its Phase III COMPACT (Clinical Studies for Optimal Management in Preventing Angioedema with low-volume subcutaneous C1-inhibitor replacement Therapy) trial showed a reduction of HAE attacks by 95 percent in patients taking 60 IU/kg Haegarda compared to placebo. Use of rescue medication was reduced by greater than 99 percent versus placebo.
The 16-week, multicenter, controlled clinical trial included 90 subjects ranging in age from 12 to 72 years old. Patients were randomized to receive twice per week subcutaneous doses of either 40 IU/kg or 60 IU/kg, and the treatment effect was compared to a placebo treatment period.
Patients in both treatment groups experienced a significantly reduced number of HAE attacks compared to their placebo treatment period.
“HAE patients face painful, debilitating, and life-disrupting swelling episodes that are also life-threatening when the airway is involved,” said Anthony J. Castaldo, President of the U.S. Hereditary Angioedema Association in a release from CLS Behring statement. “Patient care in the United States takes an important step forward with the approval of this subcutaneous treatment for preventing HAE attacks.”
The Melbourne-based biotherapeutics company plans to have Haegarda available in the U.S. soon.
Filed Under: Drug Discovery