The US Food and Drug Administration (FDA) has approved a supplemental Biologics License Application for Takeda’s lanadelumab-flyo (Takhzyro®) treatment to prevent hereditary angioedema (HAE) attacks in children aged 2 to 11 years.

“Approval of the expanded indication of Takhzyro represents a significant step forward for the HAE community as it helps some of its youngest patients who are living with the disease to have a long-term prophylaxis treatment available to them,” said Julie Kim, president, US Business Unit and US country head at Takeda.

Previously, the only prophylactic therapy for children aged 6 to 11 years required every 3 to 4 day dosing, and there was no treatment available for children under 6. With lanadelumab, the recommended dose is 150 mg/1 mL injected once every 4 weeks in children 2 to 5 years old and every 2 weeks in those aged 6 to 11 years.

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Lanadelumab is a fully human monoclonal antibody therapy designed to reduce plasma kallikrein activity, thereby preventing HAE attacks. It can be self-administered by patients aged 12 or more years and can be injected subcutaneously by a caregiver who is trained by a health professional.

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The FDA’s approval is based on efficacy data from the phase 3 HELP trial as well as from the phase 3 SPRING study on pediatric patients with HAE. The study results showed that lanadelumab reduced HAE attacks by an average of 94.8% from baseline: from 1.84 attacks per month to 0.08 attacks over a period of 1 year, with the vast majority of patients being attack-free.

Lanadelumab was approved in the US in 2018 for patients with HAE aged 12 or more years, and it is currently available in more than 60 countries around the world.


U.S. FDA approves Takeda’s TAKHZYRO® (lanadelumab-flyo) to prevent hereditary angioedema (HAE) attacks in children 2 years of age and older. News release. Takeda; February 3, 2023.