Treatment with lanadelumab (TAKHZYRO®) has demonstrated positive results in the prevention of disease activity in children aged 2 to 12 years with hereditary angioedema (HAE), according to a press release by Takeda Pharmaceuticals.

The phase 3, open-label, multicenter SPRING study was conducted in Germany and the results, which were presented at the 2022 European Academy of Allergy and Clinical Immunology Hybrid Congress, were consistent with those from earlier studies conducted in adult and adolescent patients.

Currently, no long-term prophylactic treatments have been approved for the treatment of patients with HAE less than 6 years of age. The investigators sought to assess the safety and pharmacokinetics of lanadelumab in patients with HAE between 2 and 12 years of age—the primary study objective. A secondary study objective was clinical outcomes—that is, the prevention of HAE attacks.

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In the current study, participants 2 to 6 years of age were treated with lanadelumab 150 mg every 4 weeks (q4w), whereas, in those 6 to 12 years of age, the same dose was administered to participants every 2 weeks (q2w).

“HAE is a rare condition where unpredictable symptoms like severely debilitating swelling can take a toll on children both physically and emotionally,” said Dr. Marcus Maurer, professor of Dermatology and Allergy Charité at the Universitätsmedizin Berlin in Germany, and principal investigator of the SPRING study.

Read more about the diagnosis of HAE

Attacks of HAE, which can involve serious, severely debilitating swelling in the face, throat, abdomen, feet, hands, and genitalia, may occur very early in childhood, with potentially fatal upper airway angioedema reported in individuals as young as 3 years of age. A diagnosis of HAE can take an average of 8.4 years following the onset of symptoms.

In SPRING, treatment with lanadelumab reduced the rate of HAE attacks by a mean of 94.8% vs baseline (from 1.84 attacks per month to 0.08 attacks per month). The extent of the reduction was similar between participants in the q4w and the q2w groups.

Although minimum steady-state concentrations with lanadelumab 150 mg q4w were 50% to 60% lower in patients between 2 and 6 years of age, they were nonetheless sufficient to induce a clinically meaningful treatment response. Overall, 76.2% of patients remained attack-free during the complete treatment period. A mean of 99.5% of days (range, 96.4% to 100%) was attack-free. No adverse events of special interest were reported.

“The SPRING study data reinforce the safety and efficacy of TAKHZYRO across a broad range of patients living with HAE,” Ming Yu, global clinical lead at Takeda, said.


Takeda’s TAKHZYRO® (lanadelumab) demonstrated positive results in the prevention of HAE attacks in first and only open-label phase 3 trial in children ages 2 to <12 years. News release. Takeda Pharmaceuticals; June 30, 2022.