The Food and Drug Administration (FDA) has expanded the approval of Takhzyro® (lanadelumab-flyo) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in pediatric patients 2 to less than 12 years of age. Previously, the treatment was only approved for patients 12 years of age and older.
The approval was supported extrapolation of efficacy data from the phase 3 HELP study (ClinicalTrials.gov Identifier: NCT02586805), which included patients 12 to less than 18 years of age, along with safety and pharmacodynamic data from the open-label phase 3 SPRING study (ClinicalTrials.gov Identifier: NCT04070326).
The SPRING study 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 at 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.
Findings from the SPRING study showed that treatment with lanadelumab reduced the rate of HAE attacks by a mean of 94.8% compared with baseline, from 1.84 attacks per month to 0.08 attacks during treatment. Moreover, 76.2% of patients were attack-free over the 52-week treatment period with an average of 99.5% attack-free days. No new safety signals were observed in these patients.
Takhzyro is currently supplied as a 300mg/2mL (150mg/mL) strength solution for subcutaneous injection in single-dose vials and prefilled syringes. A new single-dose prefilled syringe containing 150mg/1mL solution is expected to be available in the coming months.
- US FDA approves Takeda’s Takhzyro® (lanadelumab-flyo) to prevent hereditary angioedema (hae) attacks in children 2 years of age and older. News release. Takeda. February 3, 2023. Accessed February 6, 2023. https://www.businesswire.com/news/home/20230203005431/en/U.S.-FDA-Approves-Takeda%E2%80%99s-TAKHZYRO%C2%AE-lanadelumab-flyo-to-Prevent-Hereditary-Angioedema-HAE-Attacks-in-Children-2-Years-of-Age-and-Older.
- Takhzyro. Package insert. Takeda; 2023. Accessed February 6, 2023. https://www.shirecontent.com/PI/PDFs/TAKHZYRO_USA_ENG.pdf.