First preventive treatment for children 2 years old and up

The largest pediatric trial of any preventive treatment in HAE

This 52-week, pediatric, open-label study included 21 children diagnosed with HAE aged 2 to <12 years. The main goals of the study were to:

• Evaluate the safety of TAKHZYRO 150 mg taken once every 2 weeks or every 4 weeks
• Measure the levels of TAKHZYRO in the body for children 2 to <12 years of age

Patients and their caregivers knew they were receiving TAKHZYRO, which could have influenced the results.

In the 52-week study, there were:

• No serious side effects reported
• No discontinuations due to side effects
• No allergic reactions related to TAKHZYRO

In addition, levels of TAKHZYRO in the body for children 2 to <12 years of age who received 150 mg every 2 or 4 weeks were similar to those in adult patients receiving TAKHZYRO 300 mg every 2 weeks.

All data presented are for TAKHZYRO 300 mg every 2 weeks unless otherwise indicated.

Most common related TEAEs in children taking TAKHZYRO 150 mg every 2 or 4 weeks (21 children)

• Injection site pain: 29%
• Injection site redness: 14%
• Injection site swelling: 5%
• Administration site pain: 5%
• Injection site reaction: 5%

TEAE=treatment-emergent adverse event

TAKHZYRO may cause serious side effects, including allergic reactions. Call your healthcare provider or get emergency help right away if your child has symptoms of an allergic reaction.

The effectiveness of TAKHZYRO in children

Use of TAKHZYRO in children 2 to <12 years of age was supported by:

• Efficacy data from the 6.5-month study in people 12 years of age and older
• Additional data that showed similar levels of TAKHZYRO were reached in the body for adults and children

A secondary goal of the 52-week study was to measure the ability of TAKHZYRO 150 mg taken once every 2 weeks or once every 4 weeks to prevent HAE attacks in children 2 to <12 years of age. Decrease in HAE attacks was measured as the number of attacks before the patient started in the study compared to the number of HAE attacks after taking TAKHZYRO.

An important note about this study

The study was not designed to understand how well TAKHZYRO works in children. The 21 children included knew they were taking TAKHZYRO. This study did not compare TAKHZYRO to another product or placebo. These details make it difficult to determine how well TAKHZYRO decreased HAE attacks in children.

On average, children had:

• 95% fewer HAE attacks compared to before starting the study
• 76% of children were attack free for the entire 52-week study