FDA accepts supplemental BLA for Takhzyro in hereditary angioedema

Takeda announced that the FDA has accepted a supplemental Biologics License Application (sBLA) for the potential expanded use of Takhzyro (lanadelumab-flyo) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in pediatric patients 2 to <12 years of age. currently, children with hae under the age of 6 have no approved prophylaxis treatment. if approved, takhzyro could potentially become the first treatment of its kind for this population. the fda has granted priority review of the application and indicated a decision is expected in the first half of 2023.

The sBLA is based on data from the SPRING study, the first and only open-label Phase III trial for HAE patients under the age of 12. If approved, children in the U.S. would have access to treatment for the prevention of HAE attacks, which can involve serious and severely debilitating swelling in the abdomen, face, feet, genitals, hands and throat. Potentially fatal upper airway angioedema has been reported in patients as young as 3 years old.