FDA Accepts BLA for Takhzyro in Young Children with HAE


The FDA is expected to make a decision on approval in this patient population in the first half of 2023.

The FDA has accepted a supplemental biologics license application (sBLA) for the potential expanded use of Takeda’s Takhzyro (lanadelumab-flyo) 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 for this population. The FDA has granted priority review and indicated a decision is expected in the first half of 2023.

Takhzyro is currently approved in patients 12 years and older to prevent attacks of hereditary angioedema (HAE), a rare disease that cause recurrent episodes of swelling of the limbs, face, intestinal tract and airway. Symptoms of hereditary angioedema typically begin in childhood and worsen during puberty. On average, untreated individuals have an attack every one to two weeks, and most episodes last for about three to four days.

Hereditary angioedema is estimated to affect 1 in 50,000 people. Hereditary angioedema type I and type II is caused by mutations in the SERPING1 gene. Type III is cause mutations of the F12 gene.

Cheryl Schwartz

Cheryl Schwartz

“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,” Cheryl Schwartz, senior vice president, U.S. rare disease business unit at Takeda, said in a press release.

The sBLA is based on data from the SPRING study, a phase 3 trial for HAE patients under the age of 12. Study findings released in June 2022, indicated children ages 2 to <12 experienced 94.8% fewer hereditary angioedema attacks during treatment with Takhzyro over the 52-week treatment period compared with baseline. No deaths or serious treatment-emergent adverse events were reported during the study, and no patients withdrew from the study due to adverse events. The most commonly reported adverse event was injection site pain, and most were mild or moderate in severity. These results are consistent with the favorable efficacy and safety profile of Takhyzro observed in earlier studies with adult and adolescent patients.

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