The Food and Drug Administration (FDA) has accepted for Priority Review the supplemental Biologics License Application (sBLA) for lanadelumab-flyo for prophylaxis to prevent attacks of hereditary angioedema (HAE) in pediatric patients 2 to less than 12 years of age.
The sBLA is supported by data from the open-label, multicenter, phase 3 SPRING study (ClinicalTrials.gov Identifier: NCT04070326) that evaluated the safety and pharmacokinetics of lanadelumab to prevent acute HAE attacks in 21 patients 2 to less than 12 years of age. Lanadelumab was administered as a dose of 150mg subcutaneously every 4 weeks in patients 2 to less than 6 years of age and every 2 weeks in patients 6 to less than 12 years of age, over 52 weeks.
Findings demonstrated that treatment with lanadelumab reduced the rate of HAE attacks by a mean of 94.8% compared to baseline, from 1.84 attacks per month to 0.08 attacks during treatment. Throughout the 52-week treatment period, 76.2% (n=16) of patients were attack-free with an average of 99.5% attack-free days. The reduction in HAE attacks were similar between lanadelumab administered at a frequency of every 4 or 2 weeks.
The most commonly reported treatment-emergent adverse event (TEAE) was injection site pain, which were mild or moderate in severity. There were no deaths or serious TEAEs reported during the study, and no study discontinuations were reported due to TEAEs.
“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, US 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.”
Lanadelumab-flyo, a plasma kallikrein inhibitor (monoclonal antibody), is currently marketed under the trade name Takhzyro and is approved for prophylaxis to prevent attacks of HAE in adult and pediatric patients 12 years of age and older.
- 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. Accessed October 5, 2022. https://www.businesswire.com/news/home/20221004006135/en/U.S.-Food-and-Drug-Administration-Accepts-Takeda%E2%80%99s-Supplemental-Biologics-License-Application-for-Use-of-TAKHZYRO%C2%AE-lanadelumab-flyo-to-Prevent-Hereditary-Angioedema-HAE-Attacks-in-Children-2-Years-of-Age-and-Older
- Takeda’s Takhzyro® (lanadelumab) demonstrated positive results in the prevention of HAE attacks in first and only open-label phase 3 trial in children ages 2 to <12 years. News release. Takeda. June 30, 2022. Accessed October 5, 2022. https://www.takeda.com/newsroom/newsreleases/2022/phase-3-spring-study-data-presented-at-eaaci/