An open-label, multicenter study in patients with HAE as young as 2 years of age¹

TAKHZYRO was studied in pediatric patients 2 to <12 years of age with HAE type I or II¹

The SPRING study design

^*Eligible patients underwent a 4- to 12-week baseline observation period before initiating treatment with TAKHZYRO.¹⁰

^†Patients aged 2 to <6 years received 150 mg every 4 weeks for the 52-week treatment period.¹⁰

^‡Patients aged 6 to <12 years were to receive 150 mg every 2 weeks for 52 weeks and had an option to switch to every 4 weeks if they were attack free for 26 weeks.¹⁰

Q4W=every 4 weeks.

The co-primary endpoints of this phase 3, open-label, multicenter pediatric trial were to assess the safety and pharmacokinetics of TAKHZYRO in 21 children with HAE type I or II (2 to <12 years of age). Efficacy was a secondary endpoint of the study, specifically the normalized number of investigator-confirmed HAE attacks during treatment compared to the baseline HAE attack rate during the 4- to 12-week observation period. The treatment period was 52 weeks, divided into 26-week treatment periods A and B. In treatment period A, patients aged 2 to <6 years (n=4) and 6 to <12 years (n=17) received TAKHZYRO 150 mg every 4 weeks and 150 mg every 2 weeks, respectively. In treatment period B, patients in the 2 to <6 years age group continued receiving 150 mg every 4 weeks, while patients in the 6 to <12 years age group were permitted to switch to 150 mg every 4 weeks if they were attack free during treatment period A. Seven patients aged 6 to 12 years switched to 150 mg every 4 weeks during treatment period B, and 1 patient aged 2 to <6 years switched to 150 mg every 2 weeks during treatment period B after experiencing recurrent attacks. Patients were followed up for 2 or 4 weeks, depending on their dosing schedule.¹⁰

Established effectiveness and safety profile in pediatric patients 2 to <12 years of age¹

Co-primary Endpoint

Use of TAKHZYRO for patients 2 to <12 years of age was supported by extrapolation of efficacy data from the HELP study, with additional pharmacokinetic analyses showing similar drug exposures between adults and pediatric patients, and safety and pharmacodynamic data from the SPRING study.¹

Lanadelumab-flyo exposures in pediatric patients 2 to <12 years of age receiving TAKHZYRO 150 mg every 2 weeks or every 4 weeks were comparable to those in adult patients receiving TAKHZYRO 300 mg every 2 weeks¹⁰

• Pharmacokinetics (Co-primary Endpoint): Patients aged 2 to <12 years taking TAKHZYRO in the 52-week open-label study experienced systemic exposure to TAKHZYRO¹⁰

Secondary Endpoints

Limitations

Because this was a noncontrolled, open-label study that enrolled 21 pediatric patients and lacked statistical hypothesis testing, these data have less evidentiary value than a double-blind, placebo-controlled study. Further confirmatory studies are required to draw any conclusions from these data.¹

Patients aged 2 to <12 years taking TAKHZYRO in the 52-week open-label study experienced attack reduction vs baseline^1,10

• 95% reduction in attacks on average vs baseline (N=21)¹⁰*
  • Mean monthly attack rate at baseline (during observation period): 1.84 (N=21)¹⁰
  • Mean monthly attack rate on treatment: 0.08 (N=21)¹⁰
• 76% of patients experienced freedom from attacks for the entire 52-week study (n=16)¹⁰
• 99.5% of days on average with zero attacks during the entire treatment period (n=21)¹⁰

All data presented are for the total population of pediatric patients taking TAKHZYRO 150 mg every 2 or every 4 weeks.
*Baseline was defined as the frequency of HAE attacks in pediatric patients before they started TAKHZYRO.¹⁰

SELECT IMPORTANT SAFETY INFORMATION
Use in Specific Populations: The safety and efficacy of TAKHZYRO in pediatric patients <2 years of age have not been established.

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