The US Food and Drug Administration (FDA) has accepted a supplemental Biologics License Application for Takeda’s lanadelumab-flyo (Takhzyro®), a prophylactic treatment for hereditary angioedema (HAE) attacks in children aged 2 or more years. 

The acceptance of the application, announced via news release, means the FDA will conduct a priority review of the therapy, and a decision is anticipated in the first half of 2023.  

“Unpredictable, debilitating and potentially life-threatening HAE swelling attacks can cause a physical and emotional toll on those living with this rare disorder; this is burdensome for young children and their caregivers,” said Cheryl Schwartz, senior vice president of Takeda’s US Rare Disease Business Unit. “If Takhzyro is approved for this expanded use, children as young as 2 years old would have a treatment option which has shown proven effective prevention of HAE attacks in those over 12 years of age.”


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The application is based on the results of the SPRING study, a phase 3 open-label trial for pediatric patients 2 to 12 years of age who have HAE. In that study, the participants experienced 94.8% fewer HAE attacks over a 1-year period while being treated with 150 mg of lanadelumab-flyo every 4 weeks, compared with baseline.

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No deaths or serious adverse events were reported, and no participants withdrew from the study due to adverse events.

Lanadelumab-flyo is an injectable medicine currently approved for individuals aged 12 years or older and is available in over 30 countries. The treatment can be self-administered (with training by a healthcare professional) or administered by a caregiver. The current dosage is 300 mg/2 mL (150 mg/1 mL) every 2 or 4 weeks.

The new trial aims to determine if lanadelumab-flyo is safe and effective in children under 12 years of age; such approval would represent the world’s first and only prophylactic therapy for children under the age of 6 years with HAE.

Reference

U.S. Food and Drug Administration accepts Takeda’s supplemental Biologics License Application for use of TAKHZYRO® (lanadelumab-flyo) to prevent hereditary angioedema (HAE) attacks in children 2 years of age and older. News release. Takeda; October 5, 2022.