European Commission Approves Ruconest for Acute Swelling in Children with HAE

The European Commission has approved Pharming‘s Ruconest (conestat alfa) for the treatment of acute swelling episodes in young children with hereditary angioedema (HAE).

This approval expands the age range for Ruconest, which previously was approved to treat adults and adolescents with HAE in the European Union (EU). The medication is now authorized for use in the EU for children 2 and older.

“We are pleased to receive approval from the European Commission and to be able to offer Ruconest as a treatment for acute HAE attacks in all patients aged two years and above,” Sijmen de Vries, chief executive of Pharming, said in a press release. “This approval allows us to treat the most vulnerable patients and further demonstrates the safety and efficacy of Ruconest.”

The new approval follows a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) in late March, and was received six weeks earlier than expected. Ruconest also is an approved therapy for adults and adolescents in the U.S. and Israel, and for adults in multiple other countries.

HAE is caused by a functional lack of the protein C1 esterase inhibitor (C1-INH). The lack of this protein triggers a chain of events that causes blood vessels to widen and fluid to accumulate in tissues — in other words, swelling. The active ingredient in Ruconest — conestat alfa — is a lab-made version of C1-INH that is intended to act as a replacement for the protein in people with HAE, thereby reducing swelling.

The new approval is based on data from an open-label Phase 2 clinical trial (NCT01359969), which evaluated the safety and effectiveness of Ruconest in children  2–13 years old.

The 20 participants were treated with 50 units/kg (up to a maximum of 4200 units) of Ruconest every time they had an acute swelling attack. In total, 73 attacks were treated.

Data showed that a single dose of Ruconest was sufficient to stop all but three (95.9%) of the attacks. In those three attacks, a second dose was administered.

The trial’s primary outcome measure (endpoint) was the time from the attack to the beginning of symptom relief, which was defined as the time it took for participants to report a relative decrease of at least 20 mm on a visual analog scale (VAS) — a scale from 0 to 100 mm, with higher numbers indicating more intense symptoms.

The median time to symptom relief after Ruconest treatment was 60 minutes. The median time to minimal symptoms — where participants had no VAS scores more than 20 mm, indicative of clinical remission — was 123 minutes. Both of these values were generally consistent across attacks.

Ruconest was generally safe and well-tolerated in the study. There were no treatment-related serious adverse events (side effects) or allergic reactions, and no participants withdrew from the study because of adverse events.