The European Commission has just granted their approval to Pharming for an extended indication of RUCONEST (conestat alfa) for Hereditary Angioedema (HAE). This extension allows the therapy to be utilized for children over the age of 2. Previously, it was only approved for adults (in 2010) and adolescents (in 2016) in the European Union. This new decision to extend the indication came 6 weeks earlier than expected.

HAE

HAE patients don’t have the C1 esterase inhibitor protein.

This deficiency causes HAE attacks which are spontaneous periods of swelling throughout the body. These episodes can affect the skin, the nose, the throat, the tongue, and even the internal organs. When the swelling occurs in the airways it is particularly dangerous and can be life-threatening.

RUCONEST is a recombinant analogue of the C1 esterase inhibitor.

This protein is necessary for controlling the bodies response to infection and inflammation. Without it, the body has an excessive response. The conestat alfa in RUCONEST is essentially a version of the missing protein. It helps the body stop its overzealous response. As a result, patient’s symptoms are eased.

The new indication of this therapy, a result of the study described below, provides further evidence for both the safety and the efficacy of this treatment.

The Study

The study which led to the approval of this new indication was an open-label investigation. It aimed to determine the safety, efficacy, and pharmacokinetics of this therapy for younger patients.

Specifically, researchers evaluated a 50 U/Lg dose of RUCONEST in 20 HAE patients between the ages of 2 and 13.

In total, 73 HAE attacks were treated using the therapy. Results were assessed by a visual analogue scale which was completed by each patient/their parent.

Results:

• Median time of when relief began: 60 minutes
• Median time until patients were only experiencing minimal symptoms: 123 minutes
• 4% (3 attacks) of HAE episodes needed to be given a 2nd dose of the therapy

There were no series AEs documented from this study. The therapy was found to be generally well-tolerated and none of the participants withdrew as a result of any adverse events.

You can read more about this therapy and new indication here.