The U.S. Food & Drug Administration (FDA) accepts a supplemental Biologics License Application (sBLA) for the potential expanded use of Takeda‘s TAKHZYRO® (lanadelumab-flyo) for prophylaxis to prevent attacks of 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.
“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,” says Cheryl Schwartz, Senior Vice President, U.S. Rare Disease Business Unit at Takeda. “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.”
The sBLA is based on data from the SPRING study, the first and only open-label Phase 3 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.
Currently, TAKHZYRO is approved and available in more than 30 countries around the world and is supported by a robust clinical development program, which includes one of the largest prevention studies in HAE with the longest active treatment duration.
(Source: Takeda)
